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MA52631A - Compositions et méthodes pour le traitement de la maladie de parkinson - Google Patents

Compositions et méthodes pour le traitement de la maladie de parkinson

Info

Publication number
MA52631A
MA52631A MA052631A MA52631A MA52631A MA 52631 A MA52631 A MA 52631A MA 052631 A MA052631 A MA 052631A MA 52631 A MA52631 A MA 52631A MA 52631 A MA52631 A MA 52631A
Authority
MA
Morocco
Prior art keywords
parkinson
disease
compositions
treatment
methods
Prior art date
Application number
MA052631A
Other languages
English (en)
Inventor
Adrian Philip Kells
Bernard Ravina
Original Assignee
Voyager Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of MA52631A publication Critical patent/MA52631A/fr

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51Lyases (4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y401/00Carbon-carbon lyases (4.1)
    • C12Y401/01Carboxy-lyases (4.1.1)
    • C12Y401/01028Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
MA052631A 2018-05-15 2019-05-15 Compositions et méthodes pour le traitement de la maladie de parkinson MA52631A (fr)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201862671944P 2018-05-15 2018-05-15
US201862681891P 2018-06-07 2018-06-07
US201862684384P 2018-06-13 2018-06-13
US201862691748P 2018-06-29 2018-06-29
US201862698419P 2018-07-16 2018-07-16
US201862703137P 2018-07-25 2018-07-25
US201862741021P 2018-10-04 2018-10-04
US201862748119P 2018-10-19 2018-10-19
US201862756897P 2018-11-07 2018-11-07
US201962789909P 2019-01-08 2019-01-08
US201962831400P 2019-04-09 2019-04-09

Publications (1)

Publication Number Publication Date
MA52631A true MA52631A (fr) 2021-03-24

Family

ID=67060450

Family Applications (1)

Application Number Title Priority Date Filing Date
MA052631A MA52631A (fr) 2018-05-15 2019-05-15 Compositions et méthodes pour le traitement de la maladie de parkinson

Country Status (13)

Country Link
US (1) US12319929B2 (fr)
EP (1) EP3794126A1 (fr)
JP (1) JP2021523914A (fr)
KR (1) KR20210019996A (fr)
CN (1) CN112424359A (fr)
AU (1) AU2019268330A1 (fr)
BR (1) BR112020023082A2 (fr)
CA (1) CA3099306A1 (fr)
MA (1) MA52631A (fr)
MX (1) MX2020012077A (fr)
SG (1) SG11202011296VA (fr)
TW (1) TW202012628A (fr)
WO (1) WO2019222328A1 (fr)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
TWI744300B (zh) * 2016-03-23 2021-11-01 里爾中央醫學中心 改良性熱處理的血小板顆粒裂解液在製備用於治療神經系統疾病的組合物的用途
WO2018039375A1 (fr) 2016-08-23 2018-03-01 Akouos, Inc. Compositions et méthodes pour traiter une déficience auditive non associée à l'âge chez un sujet humain
WO2018191450A2 (fr) 2017-04-14 2018-10-18 National Taiwan University Hospital Thérapie génique pour une carence en aadc
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2021007382A1 (fr) * 2019-07-09 2021-01-14 Voyager Therapeutics, Inc. Compositions et procédés pour le traitement de la maladie de parkinson
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
CN115607684A (zh) * 2021-07-15 2023-01-17 华中科技大学 一种内耳药物纳米载体及其应用
IL311871A (en) 2021-10-08 2024-06-01 Dyno Therapeutics Inc Capsid variants and methods of using them
CN116554278A (zh) * 2022-01-30 2023-08-08 上海玮美基因科技有限责任公司 变异型腺相关病毒及其在疾病治疗中的应用

Family Cites Families (534)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3467096A (en) 1966-04-12 1969-09-16 Ferrell S Horn Multiple hypodermic syringe arrangement
BE786542A (fr) 1971-07-22 1973-01-22 Dow Corning Dispositif d'aspiration permettant d'obtenir des echantillons de cellules
US4411657A (en) 1980-05-19 1983-10-25 Anibal Galindo Hypodermic needle
US4474569A (en) 1982-06-28 1984-10-02 Denver Surgical Developments, Inc. Antenatal shunt
US5078140A (en) 1986-05-08 1992-01-07 Kwoh Yik S Imaging device - aided robotic stereotaxis system
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
US5012818A (en) 1989-05-04 1991-05-07 Joishy Suresh K Two in one bone marrow surgical needle
WO1991018088A1 (fr) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Vecteurs eucaryotiques a base de virus adeno-associes (aav)
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
GB9027917D0 (en) 1990-12-21 1991-02-13 Ici Plc Expression systems
US5199441A (en) 1991-08-20 1993-04-06 Hogle Hugh H Fine needle aspiration biopsy apparatus and method
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5387484A (en) 1992-07-07 1995-02-07 International Business Machines Corporation Two-sided mask for patterning of materials with electromagnetic radiation
AU5665694A (en) 1992-11-04 1994-05-24 Denver Biomaterials Inc. Apparatus for removal of pleural effusion fluid
FR2703253B1 (fr) 1993-03-30 1995-06-23 Centre Nat Rech Scient Applicateur d'impulsions electriques pour traitement de tissus biologiques.
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
DE69535703T2 (de) 1994-04-13 2009-02-19 The Rockefeller University AAV-vermittelte Zufuhr von DNA an Zellen des Nervensystems
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5625048A (en) 1994-11-10 1997-04-29 The Regents Of The University Of California Modified green fluorescent proteins
US5588960A (en) 1994-12-01 1996-12-31 Vidamed, Inc. Transurethral needle delivery device with cystoscope and method for treatment of urinary incontinence
WO1996017947A1 (fr) 1994-12-06 1996-06-13 Targeted Genetics Corporation Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants
EP0727187B1 (fr) 1995-02-15 2003-08-06 Joseph Eldor Aiguille spinale à ouvertures multiples
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US5741657A (en) 1995-03-20 1998-04-21 The Regents Of The University Of California Fluorogenic substrates for β-lactamase and methods of use
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US6197293B1 (en) 1997-03-03 2001-03-06 Calydon, Inc. Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof
US6676935B2 (en) 1995-06-27 2004-01-13 Cell Genesys, Inc. Tissue specific adenoviral vectors
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびaavの精製
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
WO1998010087A1 (fr) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Vecteurs d'adenovirus de chimpanze
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
ATE465267T1 (de) 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen
IL128736A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
JP2001500015A (ja) 1996-09-06 2001-01-09 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア T7ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
US20030215422A1 (en) 1996-09-11 2003-11-20 John A. Chiorini Aav4 vector and uses thereof
US5865744A (en) 1996-09-16 1999-02-02 Lemelson; Jerome H. Method and system for delivering therapeutic agents
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
ATE348155T1 (de) 1996-11-20 2007-01-15 Introgen Therapeutics Inc Ein verbessertes verfahren zur produktion und reinigung von adenoviralen vektoren
US6541258B2 (en) 1996-12-18 2003-04-01 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
US5759179A (en) 1996-12-31 1998-06-02 Johnson & Johnson Medical, Inc. Needle and valve assembly for use with a catheter
US6436708B1 (en) 1997-04-17 2002-08-20 Paola Leone Delivery system for gene therapy to the brain
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP2001517454A (ja) 1997-09-19 2001-10-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株
CA2304131A1 (fr) 1997-09-19 1999-04-01 James M. Wilson Procede de transfert de gene utilisant bcl2 et compositions utiles a cet effet
WO1999014354A1 (fr) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Procedes et produits genetiques vectoriels utiles pour obtenir un virus adeno-associe (aav)
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
NZ505325A (en) 1997-12-23 2003-07-25 Crucell Holland B Adeno-associated virus and adenovirus chimeric recombinant viruses useful for the integration of foreign genetic information into the chromosomal dna of target cells
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
WO1999043360A1 (fr) 1998-02-26 1999-09-02 The Trustees Of The University Of Pennsylvania Protection stable contre la degenerescence sarcolemmique dystrophique et retablissement du complexe sarcoglycane
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
ATE286138T1 (de) 1998-03-20 2005-01-15 Univ Pennsylvania Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren
US6521426B1 (en) 1998-04-08 2003-02-18 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. Preparation of recombinant adenovirus carrying a rep gene of adeno-associated virus
FR2778413B1 (fr) 1998-05-07 2000-08-04 Immunotech Sa Nouveaux reactifs et methode de lyse des erythrocytes
WO1999058700A1 (fr) 1998-05-11 1999-11-18 Ariad Gene Therapeutics, Inc. Compositions multivirales et utilisations de celles-ci
EP1849872A1 (fr) 1998-05-20 2007-10-31 University Of Iowa Research Foundation Vecteurs de virus adéno associés et leur utilisation
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
EP1080218A1 (fr) 1998-05-27 2001-03-07 University of Florida Procede de preparation de compositions de virus adeno-associes de recombinaison a l'aide d'un gradient d'iodixananol
CA2329143A1 (fr) 1998-05-27 1999-12-02 Cell Genesys, Inc. Expression de l'activite du facteur viii par un vecteur viral adeno-associe
CA2329259C (fr) 1998-05-27 2003-08-05 Avigen, Inc. Apport ameliore par la convection de vecteurs viraux adeno-associes (aav)
EP1621625B1 (fr) 1998-05-27 2009-05-13 Genzyme Corporation Vecteurs AAV pour la fabrication des médicaments pour l'administration amélioré par la convection
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
CA2329060C (fr) 1998-05-28 2011-09-13 John A. Chiorini Vecteurs d'aav5 et leurs utilisation
GB2338236B (en) 1998-06-13 2003-04-09 Aea Technology Plc Microbiological cell processing
GB9817662D0 (en) 1998-08-13 1998-10-07 Crocker Peter J Substance delivery
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
WO2000022152A1 (fr) 1998-10-13 2000-04-20 Avigen, Inc. Compositions et methodes de production de virus associe aux adenovirus recombine
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
EP1124976A1 (fr) 1998-10-27 2001-08-22 Crucell Holland B.V. Production amelioree de vecteurs de virus associes aux adenovirus
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6225113B1 (en) 1998-12-04 2001-05-01 Genvec, Inc. Use of trans-activation and cis-activation to modulate the persistence of expression of a transgene in an at least E4-deficient adenovirus
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6509150B1 (en) 1999-03-05 2003-01-21 Universite De Nantes Compositions and methods for recombinant Adeno-Associated Virus production
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
WO2000066780A2 (fr) 1999-04-30 2000-11-09 University Of Florida Ribozymes fournis par virus adeno-associes pour la decouverte de fonctions geniques
EP1183380A1 (fr) 1999-06-02 2002-03-06 The Trustees Of The University Of Pennsylvania Compositions et methodes pour la fabrication de virus recombines necessitant des virus auxiliaires
KR20020059370A (ko) 1999-08-20 2002-07-12 추후보정 융합 라이브러리의 제작 및 사용을 위한 방법 및 조성물
AU1071701A (en) 1999-09-29 2001-04-30 Trustees Of The University Of Pennsylvania, The Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
JP2003533170A (ja) 1999-10-07 2003-11-11 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン アデノ随伴ウイルスおよびその使用
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
AU1436401A (en) 1999-10-21 2001-05-14 Board Of Trustees Of The University Of Arkansas, The RepO-associated virus AAV REP78 major regulatory protein, mutants thereof and uses thereof
AU3642601A (en) 1999-11-05 2001-05-30 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
AU1775901A (en) 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US6613026B1 (en) 1999-12-08 2003-09-02 Scimed Life Systems, Inc. Lateral needle-less injection apparatus and method
US6726659B1 (en) 1999-12-09 2004-04-27 John E. Stocking Catheter assembly having a fenestrated dilator
EP1240345A2 (fr) 1999-12-10 2002-09-18 Ariad Gene Therapeutics, Inc. Procedes d'expression haut niveau de genes chez des primates
US6382212B1 (en) 2000-01-18 2002-05-07 Medtronic, Inc. Fenestrated surgical drape with in situ features
EP1228234A2 (fr) 2000-03-14 2002-08-07 Neurologix, Inc. Production de vecteurs de capsides chimeres
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
US6565572B2 (en) 2000-04-10 2003-05-20 Sdgi Holdings, Inc. Fenestrated surgical screw and method
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
CA2406743A1 (fr) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Vecteurs aav recombinants dotes de capsides aav5 et vecteurs aav5 pseudotypes dans des capsides heterologues
US20030013189A1 (en) 2000-04-28 2003-01-16 Wilson James M. Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
WO2001083797A2 (fr) 2000-04-28 2001-11-08 Avigen, Inc. Polynucleotides utilises dans la production de virions de virus recombinants associes aux adenovirus
DE60106763T2 (de) 2000-05-23 2006-01-26 Neurologix, Inc. Glutaminsäure decarboxylase (gad) abgabesystem zur behandlung neurodegeneraliver erkrankungen
US20020106381A1 (en) 2000-06-13 2002-08-08 High Katherine A. Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
WO2002006483A1 (fr) 2000-07-18 2002-01-24 Takeda Chemical Industries, Ltd. Nouveau peptide actif sur le plan physiologique et utilisation associee
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
DE60129229T2 (de) 2000-08-17 2008-05-08 Ozawa, Keiya Adeno-assoziierte virus-vermittelte übertragung von angiogenesefaktoren
DE10066104A1 (de) 2000-09-08 2003-01-09 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
FR2813891B1 (fr) 2000-09-14 2005-01-14 Immunotech Sa Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications
GB0024550D0 (fr) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP2002153278A (ja) 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
WO2002070719A2 (fr) 2001-01-19 2002-09-12 Trustees Of The University Of Pennsylvania Systeme d'expression genique regulable
US7588757B2 (en) 2001-03-14 2009-09-15 Genzyme Corporation Methods of treating Parkinson's disease using recombinant adeno-associated virus virions
EP1379134A4 (fr) 2001-03-14 2006-04-05 Genteric Inc Transfert de gene par mediation de virus recombine adeno-associe via une infusion retroductale de virions
US6841357B1 (en) 2001-03-30 2005-01-11 Stratagene California Method and kits for titering adeno-associated virus vectors
US6520949B2 (en) 2001-04-02 2003-02-18 Martin St. Germain Method and apparatus for administering fluid to animals subcutaneously
US7182944B2 (en) 2001-04-25 2007-02-27 The United States Of America As Represented By The Department Of Health And Human Services Methods of increasing distribution of nucleic acids
JP2004535801A (ja) 2001-05-24 2004-12-02 ジェンザイム コーポレイション 筋特異的発現ベクター
US20030096264A1 (en) 2001-06-18 2003-05-22 Psychiatric Genomics, Inc. Multi-parameter high throughput screening assays (MPHTS)
EP1409748B1 (fr) 2001-06-22 2011-10-26 The Trustees of The University of Pennsylvania Adénovirus recombinants comprenant des protéines d'adénovirus simiens et leurs utilisations
US20040136963A1 (en) 2001-06-22 2004-07-15 The Trustees Of The University Of Pennsylvania Simian adenovirus vectors and methods of use
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
EP1279740A1 (fr) 2001-07-26 2003-01-29 Vrije Universiteit Brussel Vector recombinant dérivé d'un virus-adéno-associé pour l'utilisation thérapie génique
JP2004538005A (ja) 2001-08-08 2004-12-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア シアル酸に結合するタンパク質を有するウイルスベクターの精製法
US20030092161A1 (en) 2001-09-19 2003-05-15 The Trustees Of The University Of Pennsylvania Compositions and methods for production of recombinant viruses, and uses therefor
CA2467959C (fr) 2001-11-09 2009-03-10 Robert M. Kotin Production d'un virus adeno-associe dans des cellules d'insectes
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
SG10201506627PA (en) 2001-11-13 2015-10-29 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
EP1944043A1 (fr) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Séquences d'acides aminés et d'acides nucléiques d'adénovirus simien, vecteurs les contenant, et procédés d'utilisation.
CA2990322A1 (fr) 2001-11-21 2003-06-05 The Trustees Of The University Of Pennsylvania Sequences d'acides nucleiques et d'acides amines d'adenovirus simiens, vecteurs les contenant et procedes d'utilisation associes
WO2003049764A1 (fr) 2001-12-12 2003-06-19 Fh Faulding & Co Limited Composition de preservation virale
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
JP2005516949A (ja) 2001-12-19 2005-06-09 リジュン・ワン Gdnfの骨格筋へのアデノ随伴ウイルス媒介性のデリバリー
CA2473376A1 (fr) 2002-01-16 2003-07-31 Dynal Biotech Asa Methode permettant d'isoler des acides nucleiques et des proteines contenus dans un echantillon unique
SI1504108T1 (sl) 2002-02-01 2013-07-31 Oxford Biomedica (Uk) Limited Lentiviralni vektor
GB0208390D0 (en) 2002-04-11 2002-05-22 Univ London Adeno-associated virus producer system
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
ES2280694T3 (es) 2002-04-29 2007-09-16 The Trustees Of The University Of Pennsylvania Metodo para la extraccion directa y la amplificacion de virus integrados del adn celular de tejidos.
NZ535944A (en) 2002-04-30 2007-11-30 Oncolytics Biotech Inc Viral purification method comprising a simple extraction step in which a detergent is directly added to the cell culture, thereafter, cell debris is removed from the extraction mixture by filtration or centrifugation
AU2003295312B2 (en) 2002-05-01 2008-08-14 University Of Florida Research Foundation, Inc. Improved rAAV expression systems for genetic modification of specific capsid proteins
DE60313451T2 (de) 2002-05-14 2008-01-03 Merck & Co., Inc. Verfahren zur reinigung von adenovirus
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
JP2006515162A (ja) 2002-08-29 2006-05-25 ザ ボード オブ トラスティーズ オブ ザ リーランド スタンフォード ジュニア ユニバーシティ 環状核酸ベクター、ならびに同ベクターの作製法および使用法
TW200509968A (en) 2002-11-01 2005-03-16 Elan Pharm Inc Prevention and treatment of synucleinopathic disease
EP1418185A1 (fr) 2002-11-11 2004-05-12 Aventis Pharma Deutschland GmbH Utilisation du récepteur EDG2 dans un modèle animal de crise cardiaque
US7169612B2 (en) 2002-11-11 2007-01-30 Sanofi-Aventis Deutschland Gmbh Use of EDG2 receptor in an animal model of heart failure
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US8946151B2 (en) 2003-02-24 2015-02-03 Northern Bristol N.H.S. Trust Frenchay Hospital Method of treating Parkinson's disease in humans by convection-enhanced infusion of glial cell-line derived neurotrophic factor to the putamen
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
WO2004083441A2 (fr) 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Bibliotheque peptidique aleatoire s'affichant sur des vecteurs aav
WO2004108922A2 (fr) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Procedes et compositions pour abaisser le niveaux totaux de cholesterol et traitement des maladies cardiaques
WO2005017101A2 (fr) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Virus aviaire adenoassocie (aaav) et ses utilisations
PL1625210T3 (pl) 2003-05-21 2011-05-31 Genzyme Corp Sposoby wytwarzania preparatów rekombinowanych wirionów AAV zasadniczo niezawierających pustych kapsydów
EP1486567A1 (fr) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Vecteur derivé d'un virus associé aux adenovirus pour la thérapie génique
JP4888876B2 (ja) 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
PT2657248T (pt) 2003-06-19 2017-06-26 Genzyme Corp Viriões aav com imunorreatividade diminuída e seus usos
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
WO2005001103A2 (fr) 2003-06-20 2005-01-06 The Trustees Of The University Of Pennsylvania Procede pour produire des adenovirus chimeriques et utilisations de ces derniers
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US8333734B2 (en) 2003-07-03 2012-12-18 Walter A. Zohmann Fenestrated peripheral nerve block needle and method for using the same
US7575572B2 (en) 2003-07-15 2009-08-18 Spinal Generations, Llc Method and device for delivering medicine to bone
ES2391975T3 (es) 2003-07-25 2012-12-03 Genvec, Inc. Vacunas a base de vector adenovírico
CA2533701A1 (fr) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Composes oligomeres et compositions utilisables pour moduler des petits arn non-codants
WO2005030292A2 (fr) 2003-09-26 2005-04-07 The Trustees Of The University Of Pennsylvania Methodes, compositions et dispositifs permettant d'administrer des molecules heterologues aux cellules
CN102212558B (zh) 2003-09-30 2016-08-03 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
US7135010B2 (en) 2003-09-30 2006-11-14 Damage Control Surgical Technologies, Inc. Method and apparatus for rapid deployment chest drainage
US7699852B2 (en) 2003-11-19 2010-04-20 Zimmer Spine, Inc. Fenestrated bone tap and method
WO2005056807A2 (fr) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Vecteur viral associe aux adenovirus bovins et leurs utilisations
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
EP1742668B1 (fr) 2004-04-28 2011-02-09 The Trustees of The University of Pennsylvania Liberation sequentielle de molecules immunogenes par administrations mediees par un adenovirus ou un virus adenoassocie
WO2005116224A2 (fr) 2004-05-18 2005-12-08 Children's Memorial Hospital Vecteurs viraux adeno-associes regules par la tetracycline pour l'administration de gene au systeme nerveux
DK1751275T3 (da) 2004-06-01 2017-11-27 Avigen Inc Sammensætninger og fremgangsmåder til forebyggelse af aggregering af aav-vektorer
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
DK1807009T3 (en) 2004-10-05 2015-03-02 Genzyme Corp Stair shaped needle
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
CN1286981C (zh) 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
MX2007006807A (es) 2004-12-09 2007-11-15 Genzyme Corp Expresion regulada de transgenes en el sistema nervioso central de mamiferos.
US8614101B2 (en) 2008-05-20 2013-12-24 Rapid Pathogen Screening, Inc. In situ lysis of cells in lateral flow immunoassays
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
WO2006102072A2 (fr) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Utilisation d'un polypeptide pa131 dans le traitement de l'atherosclerose
CN101203613B (zh) 2005-04-07 2012-12-12 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
PT1879624E (pt) 2005-05-02 2012-02-02 Genzyme Corp Terapia genética para distúrbios neuro-metabólicos
EP4332227B1 (fr) 2005-08-23 2026-02-11 The Trustees of the University of Pennsylvania Arn contenant des nucléosides modifiées et leurs procédés d'utilisation
WO2007024841A2 (fr) 2005-08-23 2007-03-01 The Regents Of The University Of California Canule resistant au reflux et systeme d'administration chronique d'agents therapeutique active par convection
EP1857552A1 (fr) 2006-05-20 2007-11-21 Cargill Incorporated Xylose isomérase thermostable
SI1945779T1 (sl) 2005-10-20 2013-07-31 Uniqure Ip B.V. Izboljĺ ani aav vektorji proizvedeni v celicah insekta
WO2007064739A2 (fr) 2005-11-29 2007-06-07 Surgi-Vision, Inc. Systèmes de mise en place de dérivation et/ou de localisation guidés par irm et procédés, dispositifs et programmes informatiques associés
EP1979485A2 (fr) 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Vecteurs parvoviraux auto-complémentaires et leurs procédés de fabrication et d'utilisation
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
SI2019683T2 (sl) 2006-04-25 2022-10-28 The Regents Of The University Of California Dajanje rastnih faktorjev za zdravljenje motenj CŽS
US20090317417A1 (en) 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
WO2008010864A2 (fr) 2006-04-28 2008-01-24 The Trustees Of The University Of Pennsylvania Protéine hexon d'adénovirus modifiée et ses utilisations
WO2007130519A2 (fr) 2006-05-02 2007-11-15 Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Microréseau d'acide nucléique viral et procédé d'utilisation
ES2378333T3 (es) 2006-05-25 2012-04-11 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación de genes
DK2687223T4 (da) 2006-05-30 2025-02-17 Mayo Found Medical Education & Res Påvisning og behandling af demens
TR201906106T4 (tr) 2006-06-07 2019-05-21 Genzyme Corp Amiyotrofik lateral skleroz ve diğer omurilik hastalıkları için gen tedavisi.
CN101511373B (zh) 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
EP2526774A3 (fr) 2006-07-21 2013-03-06 California Institute of Technology Administration de gène ciblée pour une vaccination des cellules dendritiques
DK2061891T3 (da) 2006-08-24 2012-07-23 Virovek Inc Ekspression i insektceller af gener med overlappende åbne læserammer, fremgangsmåder og sammensætninger hertil
WO2008067480A2 (fr) 2006-11-29 2008-06-05 Nationwide Children's Hospital Inhibition de la myostatine destinée à améliorer le muscle et/ou à améliorer la fonction musculaire
BRPI0806849A2 (pt) 2007-02-02 2015-06-23 Biogen Idec Inc Uso de semaforin 6a para a promoção de mielinização e diferenciação de oligodendrócitos
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
PL2191001T4 (pl) 2007-04-09 2017-01-31 University Of Florida Research Foundation, Inc. Kompozycje wektora RAAV mające białka kapsydu zmodyfikowane tyrozyną i sposoby ich zastosowania
WO2008128251A1 (fr) 2007-04-17 2008-10-23 The Children's Hospital Of Philadelphia Vecteur viral humanisé et procédés pour d'utilisation
BRPI0811648A2 (pt) 2007-05-17 2014-11-11 Medgenesis Therapeutix Inc Cateter para administrar um agente a partir de um suprimento para tecido visado de um organismo de mamífero, e, métodos para administrar um agente para tecido visado disposto além de tecido intermediário de um organismo de mamífero, e para administrar um agente a partir de um suprimento para tecido visado disposto além de tecido intermediário de um organismo de mamífero
EP2012122A1 (fr) 2007-07-06 2009-01-07 Medigene AG Protéines structurelles de parvovirus muté
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
EP3252161B1 (fr) 2007-06-06 2021-11-17 Genzyme Corporation Thérapie génique pour les maladies de stockage lysosomal
US8175677B2 (en) 2007-06-07 2012-05-08 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
CN101688222B (zh) 2007-06-29 2013-03-06 弗·哈夫曼-拉罗切有限公司 启动子
EP3121190A1 (fr) 2007-07-14 2017-01-25 University of Iowa Research Foundation Procédés et compositions pour traiter les maladies du cerveau
EP2019143A1 (fr) 2007-07-23 2009-01-28 Genethon Thérapie génique du CNS utilisant l'administration périphérique de vecteurs AAV
DK3093345T3 (da) 2007-07-26 2019-06-24 Uniqure Ip Bv Baculovirusvektorer omfattende gentagne kodende sekvenser med differentielle foretrukne kodoner
EP2198016B1 (fr) 2007-09-04 2015-05-27 Her Majesty The Queen In Right of Canada as represented by The Minister of Health Virus adéno-associés porcins
EP2195439B1 (fr) 2007-09-19 2013-06-19 UniQure IP B.V. Utilisation de la machinerie de réplication d'un virus adéno-associé (aav) pour une production protéique améliorée
US8099150B2 (en) 2007-09-24 2012-01-17 MRI Interventions, Inc. MRI-compatible head fixation frame with cooperating head coil apparatus
WO2009042130A2 (fr) 2007-09-24 2009-04-02 Surgivision, Inc. Systèmes et procédés d'intervention médicaux guidés par irm
US8548569B2 (en) 2007-09-24 2013-10-01 MRI Interventions, Inc. Head fixation assemblies for medical procedures
EP2058401A1 (fr) 2007-10-05 2009-05-13 Genethon Fourniture généralisée de gènes à des motoneurones utilisant l'injection périphérique de vecteurs AAV
WO2009051421A2 (fr) 2007-10-18 2009-04-23 Lg Electronics Inc. Procédé et système d'émission et de réception de signaux
EP2212348B1 (fr) 2007-11-02 2015-07-15 Cornell University Matériaux et procédés associés à une thérapie génique de troubles psychiatriques
US8340743B2 (en) 2007-11-21 2012-12-25 MRI Interventions, Inc. Methods, systems and computer program products for positioning a guidance apparatus relative to a patient
JP5758124B2 (ja) 2007-11-28 2015-08-05 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア サルサブファミリーCアデノウイルスSAdV−40、−31および−34ならびにそれらの用途
LT2220242T (lt) 2007-11-28 2017-04-10 The Trustees Of The University Of Pennsylvania Simian pošeimio b adenovirusai sadv-28,27,-29,-32,-33 ir -35 ir jų panaudojimas
PT2220241T (pt) 2007-11-28 2016-12-26 Univ Pennsylvania Adenovírus compreendendo uma proteína hexão da cápside do adenovírus e símio sadv-39 e suas utilizações
US8133488B2 (en) 2008-01-18 2012-03-13 Genentech, Inc. Methods and compositions for targeting polyubiquitin
DK2242840T3 (da) 2008-01-29 2019-10-21 Applied Genetic Tech Corporation Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension
EP3257937B1 (fr) 2008-02-19 2022-08-03 uniQure IP B.V. Optimisation de l'expression de protéines rep et cap de parvovirus dans des cellules d'insectes
WO2009136977A2 (fr) 2008-03-04 2009-11-12 The Trustees Of The University Of Pennsylvania Adénovirus simiens sadv-36, -42.1, -42.2 et -44 et leurs utilisations
WO2009120978A2 (fr) 2008-03-27 2009-10-01 The Ohio State University Traitement des troubles liés au métabolisme à l'aide du transfert de gènes hypothalamiques du bdnf et compositions associées
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
WO2009134681A2 (fr) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Vecteurs viraux aav7 pour une administration ciblée de cellules rpe
MX2010012592A (es) 2008-05-20 2011-05-05 Eos Neuroscience Inc Vectores para la administracion de proteinas sensibles a la luz y metodos de uso de las mismas.
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP2297185A1 (fr) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Capside parvovirale avec région de répétition gly-ala incorporée
US8945885B2 (en) 2008-07-03 2015-02-03 The Board Of Trustees Of The Leland Stanford Junior University Minicircle DNA vector preparations and methods of making and using the same
KR20110086553A (ko) 2008-09-29 2011-07-28 암스테르담 몰레큘러 테라퓨틱스 비. 브이. 포르포빌리노겐 디아미나아제 유전자 치료
US8940290B2 (en) 2008-10-31 2015-01-27 The Trustees Of The University Of Pennsylvania Simian adenoviruses SAdV-43, -45, -46, -47, -48, -49, and -50 and uses thereof
US20110263001A1 (en) 2008-11-14 2011-10-27 Life Technologies Corporation Compositions and Methods for Engineering Cells
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
WO2010093784A2 (fr) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Vecteurs viraux modifiés et procédés de fabrication et d'utilisation de ceux-ci
WO2010099960A2 (fr) 2009-03-04 2010-09-10 Deutsches Krebsforschungszentrum Protéine activant l'assemblage (aap) et son utilisation pour la fabrication de particules de parvovirus consistant essentiellement en vp3
AU2010227419A1 (en) 2009-03-27 2011-10-20 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
CN102439157B (zh) 2009-04-30 2015-09-16 宾夕法尼亚大学托管会 包含腺伴随病毒构建体的靶向传导气道细胞组合物
EP2952581A1 (fr) 2009-05-01 2015-12-09 Redbiotec AG Pseudo-particules de virus recombinées codées par un vecteur à multiples gènes
SG10202109219SA (en) 2009-05-02 2021-10-28 Genzyme Corp Gene therapy for neurodegenerative disorders
EP2772542B1 (fr) 2009-05-28 2016-12-28 Deutsches Krebsforschungszentrum Polypeptides de capsides AAV modifiées
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP2435559A1 (fr) 2009-05-29 2012-04-04 The Trustees Of The University Of Pennsylvania Adénovirus simien 41 et ses utilisations
CA2764176C (fr) 2009-06-16 2020-06-02 Genzyme Corporation Procedes ameliores pour la purification de vecteurs aav recombinants
WO2010148088A2 (fr) 2009-06-16 2010-12-23 Surgivision, Inc. Dispositifs guidés par irm et systèmes d'intervention guidés par irm qui peuvent suivre et générer des visualisations dynamiques des dispositifs presque en temps réel
JP2012531893A (ja) 2009-06-29 2012-12-13 ボード・オブ・リージエンツ,ザ・ユニバーシテイ・オブ・テキサス・システム アルギナーゼ製剤および方法
JP2014501097A (ja) 2009-07-06 2014-01-20 アルナイラム ファーマシューティカルズ, インコーポレイテッド 生物由来物質の産生を高めるための組成物及び方法
EP2292781A1 (fr) 2009-08-17 2011-03-09 Genethon Production des produits biopharmaceutiques à base de Baculovirus dépourvus de virions baculoviraux contaminants
WO2011038187A1 (fr) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Diversification de virus adéno-associé (aav) commandé et bibliothèques préparées à partir de ce dernier
JP5894535B2 (ja) 2009-11-09 2016-03-30 ジーンポッド セラピューティクス アーベーGenepod Therapeutics Ab invivoでのニューロン特異的な最適化された連続DOPA合成用の新規ウイルスベクター構築物
WO2011069529A1 (fr) 2009-12-09 2011-06-16 Curevac Gmbh Solution contenant du mannose pour la lyophilisation, la transfection et/ou l'injection d'acides nucléiques
WO2011088081A1 (fr) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Répétitions terminales inversées restrictives pour vecteurs viraux
US8980284B2 (en) 2010-01-25 2015-03-17 New York University Recombinant derivatives of botulinum neurotoxins engineered for trafficking studies and neuronal delivery
WO2011097456A2 (fr) 2010-02-05 2011-08-11 The University Of North Carolina At Chapel Hill Compositions et procédés d'amélioration de la transduction d'un parvovirus
WO2011101869A1 (fr) 2010-02-22 2011-08-25 Transgene Biotek Ltd. Traitement contre le cancer du foie impliquant l'adénovirus associé 2/8 et le micro-arn 101
WO2011112089A2 (fr) 2010-03-11 2011-09-15 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Séquences codant une protéine rep mutée destinées à être utilisées dans la production de vaa
EP2550021A2 (fr) 2010-03-22 2013-01-30 Association Institut de Myologie Procédés d'augmentation de l'efficacité de pénétration d'un vecteur dans un tissu cible
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011122950A1 (fr) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Vecteurs aav duplex monomériques
PL2558154T3 (pl) 2010-04-16 2020-11-30 Clearpoint Neuro, Inc. Systemy chirurgiczne MRI zawierające kaniule chirurgiczne kompatybilne z MRI do transferu substancji do i/lub od pacjenta
ES2698203T3 (es) 2010-04-23 2019-02-01 Univ Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
CA3050894C (fr) 2010-04-23 2022-10-18 University Of Massachusetts Constructions d'expression a cistrons multiples
CA2833912C (fr) 2010-04-23 2021-09-21 University Of Massachusetts Traitement a base d'aav pour des troubles lies au cholesterol
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (fr) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vecteurs et séquences pour le traitement de maladies
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
JP5010760B2 (ja) 2010-10-05 2012-08-29 タカラバイオ株式会社 ウイルスベクターの製造方法
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
ES2562421T3 (es) 2010-10-12 2016-03-04 The Children's Hospital Of Philadelphia Métodos y composiciones para tratar la hemofilia B
US10738326B2 (en) 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
US20140309172A1 (en) 2010-11-05 2014-10-16 Dagmar Ringe Ice inhibiting compounds and uses thereof
EP2637702A4 (fr) 2010-11-11 2014-11-26 Univ Miami Procédés, compositions, cellules et kits pour traiter une lésion ischémique
AU2011332025B2 (en) 2010-11-23 2015-06-25 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1321, A1325, A1295, A1309 and A1322 and uses thereof
DK2673289T3 (da) 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf
WO2012112832A1 (fr) 2011-02-17 2012-08-23 The Trustees Of The University Of Pennsylvania Compositions et procédés pour modifier une spécificité tissulaire et améliorer le transfert d'un gène induit par aav9
WO2012115980A1 (fr) 2011-02-22 2012-08-30 California Institute Of Technology Fourniture de protéines au moyen de vecteurs de virus adéno-associés (aav)
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
US20120220648A1 (en) 2011-02-24 2012-08-30 National Taiwan University Hospital Method of Treating Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Using Adeno-Associated Virus (AAV)-AADC Vector
US20140024909A1 (en) 2011-02-24 2014-01-23 MRI Interventions, Inc. Mri-guided catheters
EP2500434A1 (fr) 2011-03-12 2012-09-19 Association Institut de Myologie Vecteurs AAV sans capside, compositions et procédés pour la production des vecteurs et la thérapie génique
US9610354B2 (en) 2011-04-18 2017-04-04 National Center Of Neurology And Psychiatry Drug delivery particle and method for producing the same
EP2699688A1 (fr) 2011-04-20 2014-02-26 The Trustees Of The University Of Pennsylvania Régimes et compositions pour l'immunisation passive médiée par aav contre des agents pathogènes en suspension dans l'air
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
EP2699270B1 (fr) 2011-04-22 2017-06-21 The Regents of The University of California Virions de virus adéno-associé avec capside variant et procédés d'utilisation de ceux-ci
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
US9598468B2 (en) 2011-05-18 2017-03-21 University Of Florida Research Foundation, Incorporated Polypeptides and vectors for targeting HER2/neu expressing cells and uses thereof
US20120295960A1 (en) 2011-05-20 2012-11-22 Oxford Biomedica (Uk) Ltd. Treatment regimen for parkinson's disease
WO2012168003A1 (fr) 2011-06-06 2012-12-13 Biocartis S.A. Lyse sélective des cellules par des tensioactifs ioniques selective lysis of cells by ionic surfactants
JP6122430B2 (ja) 2011-07-27 2017-04-26 ジェネトン 改善されたバキュロウイルス発現系
EP2739341B1 (fr) 2011-08-01 2021-02-17 Alcyone Lifesciences, Inc. Dispositifs microfluidiques d'administration de médicament
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
MX349601B (es) 2011-09-08 2017-08-04 Uniqure Ip Bv Remocion de virus contaminantes de preparaciones de aav.
US9056892B2 (en) 2011-09-09 2015-06-16 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
GB201118636D0 (en) 2011-10-28 2011-12-07 Oxford Biomedica Ltd Nucleotide sequence
EP2771455B1 (fr) 2011-10-28 2016-10-05 The University of North Carolina At Chapel Hill Lignée cellulaire pour la production d'un virus adéno-associé
AU2012340567B2 (en) 2011-11-22 2017-11-23 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
WO2013078199A2 (fr) 2011-11-23 2013-05-30 Children's Medical Center Corporation Méthodes pour une administration in vivo améliorée d'arn synthétiques modifiés
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
WO2013096955A1 (fr) 2011-12-23 2013-06-27 Case Western Reserve University Modification de gène ciblée à l'aide d'un virus adéno-associé recombinant hybride
US9611305B2 (en) 2012-01-06 2017-04-04 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
PT2814513T (pt) 2012-02-14 2018-03-02 Univ California Entrega sistémica e expressão regulada de genes parácrinos para doenças cardiovasculares e outras doenças
SG11201404956PA (en) 2012-02-17 2014-09-26 Philadelphia Children Hospital Aav vector compositions and methods for gene transfer to cells, organs and tissues
PE20150163A1 (es) 2012-04-18 2015-02-23 Philadelphia Children Hospital Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav
EP2660325A3 (fr) 2012-05-02 2014-02-12 Christian Medical College Vecteurs de virus AAV et séquences de nucléotides et procédés correspondants
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
BR112014027724B1 (pt) 2012-05-08 2022-05-03 Merck Sharp & Dohme Corp. E Alectos Therapeutics Inc Composto, e, composição farmacêutica
US9677088B2 (en) 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
TWI702955B (zh) 2012-05-15 2020-09-01 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
CN105473723A (zh) 2012-05-18 2016-04-06 宾夕法尼亚大学托管会 亚家族e猿腺病毒a1302、a1320、a1331和a1337及其用途
US9192393B2 (en) 2012-05-31 2015-11-24 MRI Interventions, Inc. MRI compatible surgical drills and related methods
AU2013287261B2 (en) 2012-07-06 2019-03-07 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions
JP6093358B2 (ja) 2012-07-06 2017-03-08 タカラバイオ株式会社 アデノ随伴ウイルスベクターの産生細胞
US9031636B2 (en) 2012-07-19 2015-05-12 MRI Interventions, Inc. MRI-compatible head fixation apparatus
US9498290B2 (en) 2012-07-19 2016-11-22 MRI Interventions, Inc. Surgical navigation devices and methods
US10206693B2 (en) 2012-07-19 2019-02-19 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
BR112015002168A2 (pt) 2012-08-01 2017-11-07 Nationwide Childrens Hospital liberação intratecal de vírus 9 adeno-associado recombinante
CA2882763C (fr) 2012-08-31 2019-01-15 The United States Of America, Represented By The Secretary, Department Of Health And Human Services Transfert de gene d'aquaporine a mediation par aav pour traiter le syndrome de sjogren
US9192446B2 (en) 2012-09-05 2015-11-24 MRI Interventions, Inc. Trajectory guide frame for MRI-guided surgeries
WO2014052789A1 (fr) 2012-09-28 2014-04-03 The University Of North Carolina At Chapel Hill Vecteurs vaa ciblés sur des oligodendrocytes
US9733237B2 (en) 2012-10-31 2017-08-15 The Trustees Of Columbia University In The City Of New York Methods for identifying candidates for the treatment of neurodegenerative diseases
RU2721275C2 (ru) 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
EP2940131B1 (fr) 2012-12-25 2019-02-20 Takara Bio Inc. Variant d'aav
DK2943567T3 (en) 2013-01-08 2018-01-02 Genzyme Corp USE OF INOS INHIBITORS TO INCREASE VIRAL YIELD IN CULTURE
US9066966B2 (en) 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
WO2014124282A1 (fr) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Transfert génétique médié par aav amélioré pour thérapies rétiniennes
US10421949B2 (en) 2013-02-22 2019-09-24 Genzyme Corporation Microcarrier perfusion culturing methods and uses thereof
EP2970946A4 (fr) 2013-03-13 2016-09-07 Philadelphia Children Hospital Vecteurs viraux adéno-associés et méthodes d'utilisation associées
US9572928B2 (en) 2013-03-13 2017-02-21 MRI Interventions, Inc. Substance delivery devices, systems and methods
US9498575B2 (en) 2013-03-14 2016-11-22 MRI Interventions, Inc. Substance delivery devices, systems and methods
EP2970966A4 (fr) 2013-03-15 2016-10-26 Univ North Carolina Répétitions terminales inversées de synthèse du virus adéno-associé
US9618590B2 (en) 2013-03-15 2017-04-11 MRI Interventions, Inc. Tip assembly for MRI-compatible medical devices and MRI-compatible medical devices incorporating same
CA2904156C (fr) 2013-03-15 2023-01-10 The Children's Hospital Of Philadelphia Vecteurs comprenant des sequences polynucleotidiques de garnissage/de remplissage et leurs procedes d'utilisation
EP2968605B1 (fr) 2013-03-15 2022-07-27 The University of North Carolina At Chapel Hill Méthodes et compositions de double liaison de glycane de vecteurs avv
WO2014168953A1 (fr) 2013-04-08 2014-10-16 University Of Iowa Research Foundation Vecteur chimérique de parvovirus à virus adéno-asocié /bocavirus
EP2792742A1 (fr) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Vecteurs de thérapie génique permettant de traiter la myocardiopathie
AU2014255665B2 (en) 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
KR102413498B1 (ko) 2013-04-20 2022-06-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달
TWI500551B (zh) 2013-04-26 2015-09-21 Ful Chee Ent Co Ltd Bicycle quick release structure
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
AU2013388083B2 (en) 2013-05-01 2019-08-22 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
SG11201509419QA (en) 2013-05-15 2015-12-30 Univ Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
WO2014186746A1 (fr) 2013-05-16 2014-11-20 University Of Florida Research Foundation, Inc. Éléments d'arnm en épingle à cheveux et procédés de régulation de la traduction des protéines
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
WO2014201308A1 (fr) 2013-06-12 2014-12-18 Washington University Vecteurs adénoviraux ciblant les cellules endothéliales et procédés et utilisations associés
EP3567112A1 (fr) 2013-06-13 2019-11-13 Translate Bio, Inc. Production virale basée sur l'arn messager
PH12016500162B1 (en) 2013-07-22 2024-02-21 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues
CN105764532B (zh) 2013-07-26 2021-08-31 衣阿华大学研究基金会 治疗脑疾病的方法和组合物
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
US11078464B2 (en) 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
LT3044231T (lt) 2013-09-12 2020-11-10 Biomarin Pharmaceutical Inc. Aav vektoriai, apimantys viii faktorių koduojantį geną
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
JP6985795B2 (ja) 2013-09-26 2021-12-22 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー
US20160354489A1 (en) 2013-09-26 2016-12-08 Universitat Autònome de Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
PT3459965T (pt) 2013-10-11 2021-02-25 Massachusetts Eye & Ear Infirmary Métodos de previsão de sequências de vírus ancestrais e suas utilizações
WO2015057807A1 (fr) 2013-10-16 2015-04-23 MRI Interventions, Inc. Systèmes chirurgicaux de navigation, dispositifs et méthodes associés
JP6873699B2 (ja) 2013-10-24 2021-05-19 ユニキュアー アイピー ビー.ブイ. Aav5カプシドタンパク質を含むアデノ随伴ウイルス(aav)を用いた神経学的疾患の処置
US10087224B2 (en) 2013-11-01 2018-10-02 Cornell University Gene therapy for Alzheimer's and other neurodegenerative diseases and conditions
EP3066195A4 (fr) 2013-11-08 2017-06-28 The Board of Trustees of the University of Arkansas Oncogène "x" de virus adéno-associés
US9644216B2 (en) 2013-11-26 2017-05-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Adeno-associated virus vectors for treatment of glycogen storage disease
US10161011B2 (en) 2013-11-29 2018-12-25 Takara Bio Inc. Method for quantifying adeno-associated virus
AU2014363988B2 (en) 2013-12-09 2020-06-11 Baylor College Of Medicine Hippo and dystrophin complex signaling in cardiomyocyte renewal
AU2014362245A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
ES2732208T3 (es) 2014-01-09 2019-11-21 Genethon Métodos para el diagnóstico o el seguimiento de distrofias musculares
WO2015106273A2 (fr) 2014-01-13 2015-07-16 Trustees Of Boston University Méthodes et dosages concernant la chorée de huntington et la maladie de parkinson
GB201401707D0 (en) 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
US10595744B2 (en) 2014-02-14 2020-03-24 MRI Interventions, Inc. Surgical tool-positioning devices and related methods
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
KR20160122161A (ko) 2014-02-19 2016-10-21 에프. 호프만-라 로슈 아게 혈액 뇌 장벽 셔틀
WO2015127128A2 (fr) 2014-02-19 2015-08-27 University Of Massachusetts Vaa recombinants ayant des propriétés de transcytose utiles
WO2015124546A1 (fr) 2014-02-19 2015-08-27 Fundación Centro Nacional De Investigaciones Cardiovasculares Carlos Iii- Cnic Vecteurs aav destinés au traitement des cardiopathies ischémiques et non ischémiques
WO2015126972A1 (fr) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Procédés et compositions pour l'administration de gènes à des cellules bipolaires on
US10272117B2 (en) 2014-02-24 2019-04-30 Celgene Corporation Methods of using an activator of cereblon for neural cell expansion and the treatment of central nervous system disorders
WO2015131076A1 (fr) 2014-02-27 2015-09-03 Yale University Sécrétion de mucus dans l'épithélium intestinal régulée par l'inflammasome nlrp6
ES2768763T3 (es) 2014-03-04 2020-06-23 Univ Florida Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR
WO2015134920A1 (fr) 2014-03-07 2015-09-11 The Arizona Board Of Regents On Behalf Of The University Of Arizona Peptides crmp2 non narcotiques ciblant des canaux sodiques pour la douleur chronique
ES2821938T3 (es) 2014-03-09 2021-04-28 Univ Pennsylvania Composiciones útiles en el tratamiento de la deficiencia de ornitina transcarbamilasa (OTC)
KR102572449B1 (ko) 2014-03-10 2023-08-31 유니큐어 아이피 비.브이. 곤충 세포에서 생산된 더욱 향상된 aav 벡터
US20170114346A1 (en) 2014-04-03 2017-04-27 Braingene Ab Gene expression system and regulation thereof
EP3129485B2 (fr) 2014-04-09 2022-12-21 Editas Medicine, Inc. Méthodes et compositions se rapportant à crispr/cas et destinées au traitement de la fibrose kystique
EP2933335A1 (fr) 2014-04-18 2015-10-21 Genethon Procédé de traitement de neuropathies périphériques et sclérose latérale amyotrophique
EP3134522B1 (fr) 2014-04-25 2021-10-06 University of Massachusetts Vecteurs de virus adéno-associés recombinants utiles pour réduire une immunité contre des produits transgéniques
BR112016024379A2 (pt) 2014-04-25 2017-10-10 Univ Pennsylvania variantes ldlr e seu uso em composições para reduzir os níveis de colesterol
CA2946593A1 (fr) 2014-05-02 2015-11-05 Genzyme Corporation Vecteurs aav pour therapie genique de la retine et du snc
WO2015173436A1 (fr) 2014-05-16 2015-11-19 Vrije Universiteit Brussel Correction génétique d'une dystrophie myotonique de type 1
EP3151866B1 (fr) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Capsides chimériques
US11207424B2 (en) 2014-06-13 2021-12-28 Mayo Foundation For Medical Education And Research Methods and materials for increasing viral vector infectivity
WO2015196179A1 (fr) 2014-06-20 2015-12-23 University Of Florida Research Foundation, Inc. Procédés d'encapsulation de plusieurs vecteurs viraux associés aux adénovirus
EP2960336A1 (fr) 2014-06-27 2015-12-30 Genethon Traitement systémique efficace de pathologies dystrophiques musculaires
WO2016004319A1 (fr) 2014-07-02 2016-01-07 University Of Florida Research Foundation, Inc. Compositions et procédés pour purifier un virus adéno-associé recombinant
WO2016006658A1 (fr) 2014-07-10 2016-01-14 タカラバイオ株式会社 Procédé de fabrication de particules de virus sans enveloppe
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
US10392622B2 (en) 2014-08-01 2019-08-27 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression
PL3198018T3 (pl) 2014-09-24 2021-07-19 City Of Hope Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3795580A1 (fr) 2014-10-03 2021-03-24 University of Massachusetts Vecteurs aav identifiés au moyen de banques à efficacité élevée
US10842886B2 (en) 2014-10-10 2020-11-24 Research Institute At Nationwide Children's Hospital Guided injections for AAV gene transfer to muscle
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA2966884A1 (fr) 2014-11-05 2016-05-12 Research Institute At Nationwide Children's Hospital Procedes et materiels pour la production de virus recombines dans des microalgues eucaryotes
SI3221349T1 (sl) 2014-11-19 2021-02-26 Axon Neuroscience Se Humanizirano tau protitelo pri alzheimerjevi bolezni
CN114480440A (zh) 2014-11-21 2022-05-13 北卡罗来纳-查佩尔山大学 靶向中枢神经系统的aav载体
US10900053B2 (en) 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
DK3591070T3 (da) 2014-11-28 2023-01-30 Uniqure Ip Bv DNA-forureninger i en sammensætning omfattende en parvovirusvirion
WO2016094783A1 (fr) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions et méthodes pour la production de scaav
EP3245220B1 (fr) 2015-01-14 2023-09-20 The University of North Carolina at Chapel Hill Procédés et compositions destinés au transfert de gènes ciblé
CA2975447C (fr) 2015-01-30 2021-02-23 The Regents Of The University Of California Systeme de delivrance de genes sous-pial spinal
WO2016126857A1 (fr) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Mutants de recombinaison aav1, aav5 et aav6 et leurs utilisations
IL296466B2 (en) 2015-02-10 2024-11-01 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
SG10201907396XA (en) 2015-02-10 2019-09-27 Genzyme Corp VARIANT RNAi
US20180135074A1 (en) * 2015-02-19 2018-05-17 University Of Florida Research Foundation, Incorporated Recombinant aav vectors for gene therapy of human hematopoietic disorders
KR102655319B1 (ko) 2015-02-20 2024-04-04 유니버시티 오브 아이오와 리써치 파운데이션 유전적 안 질환 치료용 방법 및 조성물
EP3262162A4 (fr) 2015-02-23 2018-08-08 Voyager Therapeutics, Inc. Expression régulable au moyen du virus adéno-associé (vaa)
EP3268024B1 (fr) 2015-03-10 2020-09-09 The Trustees of Columbia University in the City of New York Constructions de vecteur viral adéno-associé glut1 de recombinaison et procédés associés permettant de restaurer l'expression de glut1
CN107614008A (zh) 2015-03-20 2018-01-19 蓝鸟生物公司 载体制剂
CN107532177A (zh) 2015-03-24 2018-01-02 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
WO2016161388A1 (fr) 2015-04-03 2016-10-06 University Of Massachusetts Petit arn interférent asymétrique entièrement stabilisé
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3626274B1 (fr) 2015-04-16 2025-01-22 Emory University Promoteurs recombinants et vecteurs pour l'expression de protéines dans le foie et leur utilisation
WO2016172155A1 (fr) 2015-04-23 2016-10-27 University Of Massachusetts Modulation de l'expression d'un transgène du vecteur aav
CA3021949C (fr) 2015-04-24 2023-10-17 University Of Massachusetts Constructions d'aav modifiees et utilisations de celles-ci
US20160346359A1 (en) 2015-05-01 2016-12-01 Spark Therapeutics, Inc. Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease
WO2016179496A1 (fr) 2015-05-07 2016-11-10 Massachusetts Eye And Ear Infirmary Procédés d'administration d'un agent à l'oeil
EP3294398A4 (fr) 2015-05-11 2019-01-09 Alcyone Lifesciences, Inc. Systèmes et procédés d'administration de médicaments
EP3294894B8 (fr) 2015-05-12 2019-09-25 The U.S.A. as represented by the Secretary, Department of Health and Human Services Isolat de virus adéno-associé et protéine de fusion comprenant un peptide signal du facteur de croissance des nerfs et l'hormone parathyroïdienne
US20170067028A1 (en) 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
WO2016191418A1 (fr) 2015-05-26 2016-12-01 Salk Institute For Biological Studies Vecteurs d'expression spécifiques de motoneurones
US10214731B2 (en) 2015-05-28 2019-02-26 Cornell University Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema
IL302965B2 (en) 2015-05-29 2025-12-01 Univ Pennsylvania Compositions and methods for degradation of misfolded proteins
US11027024B2 (en) 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
PL3313991T3 (pl) 2015-06-23 2024-11-04 The Children's Hospital Of Philadelphia Modyfikowany czynnik ix oraz kompozycje, sposoby i zastosowania do transferu genów do komórek, narządów i tkanek
WO2017004514A1 (fr) 2015-07-02 2017-01-05 University Of Florida Research Foundation, Inc. Vecteurs de virus adéno-associé recombinant vers un carcinome médullaire de la thyroïde cible
US20170007669A1 (en) 2015-07-07 2017-01-12 Mayo Foundation For Medical Education And Research Peptide-mediated delivery of active agents across the blood-brain barrier
US10801040B2 (en) 2015-07-07 2020-10-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject
WO2017015102A1 (fr) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions et procédés pour obtenir des niveaux élevés de transduction dans des cellules hépatiques humaines
CA2988471A1 (fr) 2015-07-28 2017-02-02 University Of Massachusetts Expression transgenique de dnase i in vivo fournie par un vecteur de virus adeno-associe
HUE061388T2 (hu) 2015-07-30 2023-06-28 Massachusetts Eye & Ear Infirmary Õsi vírusszekvenciák és alkalmazásaik
WO2017023724A1 (fr) * 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions et méthodes pour le traitement d'une déficience en aadc
AU2016302335B2 (en) 2015-08-06 2022-08-04 The Trustees Of The University Of Pennsylvania GLP-1 and use thereof in compositions for treating metabolic diseases
PE20181338A1 (es) 2015-09-24 2018-08-21 Biomarin Pharm Inc Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden
IL292830B2 (en) 2015-09-28 2023-06-01 Univ Florida Methods and compositions for stealth virus antibody vectors
ES3008383T3 (en) 2015-10-09 2025-03-24 Genzyme Corp Improved flare (flow cytometry attenuated reporter expression) technology for rapid bulk sorting
US10123969B2 (en) 2015-10-15 2018-11-13 Wisconsin Alumni Research Foundation Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid
WO2017066764A2 (fr) 2015-10-16 2017-04-20 William Marsh Rice University Modification de la région n-terminale des protéines de capsides pour améliorer les propriétés des virus adéno-associés
ES2978086T3 (es) 2015-10-22 2024-09-05 Univ Massachusetts Terapia génica con aspartoacilasa en el tratamiento de enfermedad de Canavan
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
CA3002980A1 (fr) 2015-10-22 2017-04-27 University Of Massachusetts Vecteurs du serotype de virus adeno-associes ciblant la prostate
CN108601771B (zh) 2015-10-23 2023-02-21 衣阿华大学研究基金会 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法
EP3368054A4 (fr) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. Expression régulable au moyen d'un virus adéno-associé (vaa)
AU2016344065A1 (en) 2015-10-28 2018-05-10 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
JP6998869B2 (ja) 2015-11-08 2022-02-04 ジェネンテック, インコーポレイテッド 多重特異性抗体のスクリーニング方法
WO2017083423A1 (fr) 2015-11-10 2017-05-18 The Board Of Trustees Of The Leland Stanford Junior University Procédés et compositions pour moduler une infection par le vaa
EP3374494A4 (fr) 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. Compositions crispr et leurs méthodes d'utilisation pour la thérapie génique
US10240145B2 (en) 2015-11-25 2019-03-26 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas-mediated genome editing to treat EGFR-mutant lung cancer
CN108603174A (zh) 2015-12-01 2018-09-28 星火治疗有限公司 在适用于临床应用的无血清悬浮细胞培养体系中产生重组腺相关病毒(aav)载体的可扩展方法
WO2017095753A1 (fr) 2015-12-01 2017-06-08 Invivo Therapeutics Corporation Compositions et procédés de préparation d'un milieu injectable à administrer dans le système nerveux central
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
WO2017093330A1 (fr) 2015-12-03 2017-06-08 Genethon Compositions et procédés permettant d'améliorer l'efficacité de vecteurs viraux
WO2017100674A1 (fr) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Procédé de purification évolutif pour virus adéno-associé (aav) 1
EP3387118B1 (fr) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Procédé de purification évolutif d'aavrh10
EP4215605A1 (fr) 2015-12-11 2023-07-26 The Trustees of The University of Pennsylvania Procédé de purification évolutif d'aav8
EP3387137B1 (fr) 2015-12-11 2021-02-03 California Institute of Technology Peptides de ciblage pour diriger des virus adéno-associés (aav)
EP3387138B1 (fr) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Méthode de purification évolutive pour virus adéno-associé 9 (aav9)
US11124546B2 (en) 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
US11208630B2 (en) 2015-12-24 2021-12-28 University Of Florida Research Foundation, Incorporated AAV production using suspension adapted cells
EP3769788A1 (fr) 2016-01-13 2021-01-27 Genzyme Corporation Vecteurs aav recombinés exprimant des gènes ostéoprotecteurs, notamment has2 et lubricine, utiles dans le traitement de l'arthrose et des pathologies articulaires chez les mammifères
EP3403675B1 (fr) 2016-01-15 2021-04-21 Jichi Medical University Virion viral adéno-associé pour son utilisation dans le traitement de l'épilepsie
CA3011939A1 (fr) 2016-02-02 2017-08-10 University Of Massachusetts Procede pour ameliorer l'efficacite de l'administration au systeme nerveux central d'un gene aav par voie systemique
SI3411484T1 (sl) 2016-02-05 2023-12-29 Emory University Injiciranje enojnega ali samokomplementarnega adeno-povezanega virusa 9 v cerebrospinalno tekočino
WO2017139381A1 (fr) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Procédés pour produire des virus adéno-associés/bocavirus parvovirus chimériques
CN109415704B (zh) 2016-02-16 2022-02-25 利兰斯坦福初级大学董事会 对先已存在的人中和抗体有抗性的新型重组腺相关病毒衣壳
US20190071681A1 (en) 2016-02-26 2019-03-07 University Of Florida Research Foundation, Incorporated Aav heparin mutants that display significantly improved eye and brain transduction
CA3016314A1 (fr) 2016-03-02 2017-09-08 Julianne REIDERS Therapie pour demence frontotemporale
JP6994140B2 (ja) 2016-03-03 2022-02-03 ユニバーシティ オブ マサチューセッツ 非ウイルス的遺伝子導入のための閉鎖型直鎖状二重鎖dna
CA3016985C (fr) 2016-03-07 2023-07-04 University Of Iowa Research Foundation Expression mediee par aav utilisant un promoteur et un activateur synthetiques
AU2017237912B2 (en) 2016-03-23 2022-03-10 The Regents Of The University Of California Methods of treating mitochondrial disorders
WO2017165859A1 (fr) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Protéines de capside virale modifiées
AU2017245099B2 (en) 2016-03-28 2021-07-29 The Regents Of The University Of California Anti-Ryk antibodies and methods of using the same
FI3436593T3 (fi) 2016-03-28 2023-03-16 Ultragenyx Pharmaceutical Inc Adenoviruksen lämpöinaktivointimenetelmiä
CA3019126A1 (fr) 2016-03-30 2017-10-05 Spark Therapeutics, Inc. Lignee cellulaire pour la production de proteine recombinante et/ou d'un vecteur viral recombinant
RU2754467C2 (ru) 2016-03-31 2021-09-02 Спарк Терапьютикс, Инк. ПОЛНОСТЬЮ МАСШТАБИРУЕМЫЙ СПОСОБ ПРОИЗВОДСТВА rAAV НА ОСНОВЕ КОЛОНОК
IL298604A (en) 2016-04-15 2023-01-01 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
KR20250065734A (ko) 2016-04-16 2025-05-13 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 바큘로바이러스 시스템-생산된 재조합 아데노-연관 바이러스의 생물학적 효력을 증진시키는 방법
NZ744418A (en) 2016-04-21 2025-10-31 Virovek Inc Aav production in insect cells, methods and compositions therefor
EP3235516B1 (fr) 2016-04-22 2019-06-26 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts Universitätsmedizin Vecteur de virus adéno-associés (aav) régulable
WO2017190031A1 (fr) 2016-04-28 2017-11-02 Indiana University Research And Technology Corporation Procédés et compositions permettant de dédoubler les composants d'une préparation virale
WO2017192699A1 (fr) 2016-05-03 2017-11-09 Children's Medical Research Institute Polynucléotides, polypeptides et virions de virus adéno-associé
WO2017192750A1 (fr) 2016-05-04 2017-11-09 Oregon Health & Science University Vecteurs viraux adéno-associés recombinés
WO2018191450A2 (fr) 2017-04-14 2018-10-18 National Taiwan University Hospital Thérapie génique pour une carence en aadc
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3784780A1 (fr) * 2018-04-27 2021-03-03 Voyager Therapeutics, Inc. Procédés de mesure de la puissance de vecteurs viraux aadc
EP4097239A2 (fr) * 2020-01-29 2022-12-07 Voyager Therapeutics, Inc. Procédés et systèmes de production de particules d'aav

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