[go: up one dir, main page]

MA51353B1 - Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit - Google Patents

Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

Info

Publication number
MA51353B1
MA51353B1 MA51353A MA51353A MA51353B1 MA 51353 B1 MA51353 B1 MA 51353B1 MA 51353 A MA51353 A MA 51353A MA 51353 A MA51353 A MA 51353A MA 51353 B1 MA51353 B1 MA 51353B1
Authority
MA
Morocco
Prior art keywords
aavrh74
aav9
associated virus
hybrid
recombinant adeno
Prior art date
Application number
MA51353A
Other languages
English (en)
Other versions
MA51353A1 (fr
Inventor
Federico Mingozzi
Giuseppe Ronzitti
Isabelle Richard
Evelyne Gicquel
Patrice Vidal
Original Assignee
Univ Sorbonne
Genethon
Inst Nat Sante Rech Med
Univ Devry Val Dessonne
Association Inst De Myologie
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 Univ Sorbonne, Genethon, Inst Nat Sante Rech Med, Univ Devry Val Dessonne, Association Inst De Myologie filed Critical Univ Sorbonne
Publication of MA51353A1 publication Critical patent/MA51353A1/fr
Publication of MA51353B1 publication Critical patent/MA51353B1/fr

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/075—Adenoviridae
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)

Abstract

L'invention concerne une protéine de la capside du virus adéno-associé (aav) recombinant, qui est un hybride entre le sérotype 9 d'aav (aav9) et des protéines de la capside de sérotype 74 d'aav (aavrh74), ladite protéine de la capside d'aav hybride recombinante présentant un tropisme hépatique réduit par rapport aux protéines de la capside d'aav9 et d'aavrh74 parents. L'invention concerne également les particules de vecteur de sérotype d'aav hybrides dérivées qui conditionnent un gène d'intérêt et leur utilisation en thérapie génique, en particulier pour le traitement de maladies génétiques neuromusculaires.
MA51353A 2018-04-05 2019-04-04 Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit MA51353B1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18305399 2018-04-05
PCT/EP2019/058560 WO2019193119A1 (fr) 2018-04-05 2019-04-04 Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

Publications (2)

Publication Number Publication Date
MA51353A1 MA51353A1 (fr) 2021-09-30
MA51353B1 true MA51353B1 (fr) 2022-09-30

Family

ID=62063468

Family Applications (1)

Application Number Title Priority Date Filing Date
MA51353A MA51353B1 (fr) 2018-04-05 2019-04-04 Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

Country Status (14)

Country Link
US (3) US12037362B2 (fr)
EP (2) EP3775173A1 (fr)
JP (2) JP7374119B2 (fr)
KR (2) KR20210015770A (fr)
CN (2) CN112004925B (fr)
AU (2) AU2019249890B2 (fr)
BR (2) BR112020020223A2 (fr)
CA (2) CA3093347A1 (fr)
IL (2) IL277740A (fr)
MA (1) MA51353B1 (fr)
NZ (1) NZ767516A (fr)
TN (1) TN2020000187A1 (fr)
WO (2) WO2019193119A1 (fr)
ZA (1) ZA202005422B (fr)

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112040988A (zh) * 2018-04-27 2020-12-04 海德堡大学 修饰的aav衣壳多肽用于治疗肌肉疾病
CN114521143B (zh) 2019-09-19 2025-03-14 吉尼松公司 减轻fkrp心脏毒性的基因治疗表达系统
EP4073087A4 (fr) * 2019-11-08 2023-11-29 The Board Of Regents Of The University Of Texas System Vecteur viral adéno-associé recombiné pour la délivrance de gènes
EP4073257A1 (fr) * 2019-12-09 2022-10-19 UCL Business Ltd Composition de thérapie génique et traitement de cardiomyopathie liée à la myh7
CA3156611A1 (fr) 2019-12-18 2021-06-24 Serge Braun Production de vecteurs viraux recombines a partir de racines velues de plantes
WO2021229255A1 (fr) 2020-05-14 2021-11-18 Genethon Outils et procédé de prévention d'une neutralisation des virus vaa par des anticorps
EP3913060A1 (fr) 2020-05-22 2021-11-24 Genethon Vecteurs codant la glucose-6-phosphatase (g6pase-a) pour la thérapie génique
EP4162049A1 (fr) 2020-06-09 2023-04-12 Genethon Inhibiteurs de cilp-1 destinés à être utilisés dans le traitement de cardiomyopathies dilatées
US20230265427A1 (en) 2020-06-09 2023-08-24 Genethon Treatment of Genetic Dilated Cardiomyopathies
US20230250450A1 (en) 2020-06-09 2023-08-10 Genethon NRF2 Activator for Use in Treating Dilated Cardiomyopathies
CA3182313A1 (fr) 2020-06-19 2021-12-23 Isabelle Richard Systeme d'expression de therapie genique permettant une expression adequate dans les muscles et dans le c?ur de sgcg
EP4176064A1 (fr) 2020-07-03 2023-05-10 Genethon Procédé d'ingénierie de nouveaux capsides aav hybrides par l'intermédiaire d'une permutation de régions hypervariables
CN116194587A (zh) 2020-07-10 2023-05-30 吉尼松公司 一种新的肌肉特异性启动子
JP2023544683A (ja) 2020-09-10 2023-10-25 ジェネトン ペプチド改変されたａａｖカプシド
CN116323941A (zh) 2020-09-29 2023-06-23 吉尼松公司 通过诱导抗肌萎缩蛋白相关蛋白调节元件内的突变增强细胞中抗肌萎缩蛋白相关蛋白表达及其治疗用途
EP4273236A4 (fr) * 2020-12-29 2025-01-08 Jiangsu Genscript Probio Biotech Co Ltd Souche de cellule hek293t ayant une dispersibilité élevée et son procédé de criblage
US20240165271A1 (en) 2021-03-26 2024-05-23 The Board Of Regents Of The University Of Texas System Nucleotide editing to reframe dmd transcripts by base editing and prime editing
WO2022226301A1 (fr) * 2021-04-23 2022-10-27 The University Of North Carolina At Chapel Hill Capsides de vaa tropiques chimériques cardiaques
WO2023012313A1 (fr) 2021-08-04 2023-02-09 Genethon Promoteurs hybrides pour l'expression génique dans les muscles et dans le snc
CN114276419B (zh) * 2021-12-30 2023-11-17 上海勉亦生物科技有限公司 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
KR20240137067A (ko) 2022-01-25 2024-09-19 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 개선된 심장 형질도입 및 간의 탈표적화를 위한 aav 캡시드
CN114657152A (zh) * 2022-03-31 2022-06-24 苏州博腾生物制药有限公司 一种新型aav血清型及其制备方法
CN119343446A (zh) 2022-06-09 2025-01-21 吉尼松公司 用于治疗糖原贮积病iii的n-端截短gde
CN119403823A (zh) 2022-06-10 2025-02-07 吉尼松公司 具有增强的肌肉转导效率的肽修饰的aav衣壳
WO2024074727A1 (fr) 2022-10-07 2024-04-11 Genethon Immunothérapie cellulaire par car-t anti-fap pour traiter des myopathies squelettiques
WO2024079249A1 (fr) 2022-10-12 2024-04-18 Genethon Vecteur aav hybride améliorant l'expression d'un transgène dans le foie
CN116041443B (zh) * 2022-12-30 2023-09-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024173802A2 (fr) * 2023-02-17 2024-08-22 Biogen Ma Inc. Peptides contenant rgd à des fins de distribution de charges utiles
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024188913A1 (fr) 2023-03-10 2024-09-19 Genethon Induction de la tolérance immunitaire par un vecteur aav comprenant la combinaison d'une capside ciblant le foie et d'un promoteur tandem spécifique du foie et des muscles
WO2025003477A1 (fr) 2023-06-29 2025-01-02 Genethon Enzymes de débranchement du glycogène tronquées n-terminales pour le traitement de la glycogénose de type iii
WO2025015036A1 (fr) * 2023-07-10 2025-01-16 Kate Therapeutics, Inc. Approche de thérapie génique par vaa pour le traitement de la lgmd2b/dysferlinopathie
WO2025017168A1 (fr) 2023-07-19 2025-01-23 Genethon Nouveaux micro-gènes d'utrophine optimisés
WO2025017169A1 (fr) 2023-07-20 2025-01-23 Genethon Nouvelles mididystrophines
CN118005749B (zh) * 2023-07-21 2024-07-16 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN117031014A (zh) * 2023-08-14 2023-11-10 陕西脉元生物科技有限公司 抗matr3自身抗体的试剂在制备检测神经系统自身免疫疾病的产品中的应用及试剂盒

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2345731T3 (en) 2003-09-30 2016-01-25 Univ Pennsylvania Adeno-associated virus (AAV) groupings, sequences, vectors containing the same and uses thereof
EP2359867B1 (fr) 2005-04-07 2014-10-08 The Trustees of The University of Pennsylvania Procédé d'amélioration de la fonction d'un vecteur AAV
DK2675902T3 (da) 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
WO2013123503A1 (fr) 2012-02-17 2013-08-22 The Children's Hospital Of Philadelphia Compositions de vecteurs de virus adéno-associés et méthodes de transfert de gènes dans des cellules, des organes et des tissus
KR102063483B1 (ko) 2012-04-18 2020-01-08 더 칠드런스 호스피탈 오브 필라델피아 Ａａｖ 캡시드 변이체를 이용한 고효율 유전자 전달을 위한 조성물 및 방법
EP2660325A3 (fr) 2012-05-02 2014-02-12 Christian Medical College Vecteurs de virus AAV et séquences de nucléotides et procédés correspondants
CN105612253A (zh) * 2013-03-15 2016-05-25 费城儿童医院 含有填充者/填充物多核苷酸序列的载体及其制备方法
EA032706B1 (ru) * 2013-04-20 2019-07-31 Рисёрч Инститъют Эт Нэйшнвайд Чилдрен'С Хоспитал ДОСТАВКА НАЦЕЛЕННЫХ НА ЭКЗОН 2 ПОЛИНУКЛЕОТИДНЫХ КОНСТРУКЦИЙ U7snRNA ПРИ ПОМОЩИ РЕКОМБИНАНТНОГО АДЕНОАССОЦИИРОВАННОГО ВИРУСА
KR102445330B1 (ko) 2013-07-12 2022-09-19 더 칠드런스 호스피탈 오브 필라델피아 Ａａｖ 벡터 및 항―ａａｖ （아데노-관련 바이러스） 중화 항체에 대한 검정
RU2697444C2 (ru) 2013-07-22 2019-08-14 Дзе Чилдрен'З Хоспитал Оф Филадельфия Вариант aav, композиции и способы, в которых он используется, а также способы его применения для переноса генов в клетки, органы и ткани
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
BR112017005892A2 (pt) 2014-09-24 2017-12-12 Hope City variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
EP3384034B1 (fr) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Nouveaux capsides de virus adéno-associés recombinants avec du tropisme pour les muscles sqeletiques humains amélioré
US11155817B2 (en) 2016-03-18 2021-10-26 The Children's Hospital Of Philadelphia Therapeutic for treatment of diseases including the central nervous system
SG11201811603WA (en) 2016-07-26 2019-02-27 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
CA3094465A1 (fr) * 2018-04-03 2019-10-10 Stridebio, Inc. Vecteurs de virus permettant de cibler des tissus ophtalmiques

Also Published As

Publication number Publication date
CA3134183A1 (fr) 2020-10-08
WO2020200499A1 (fr) 2020-10-08
KR20210015770A (ko) 2021-02-10
AU2019249890A1 (en) 2020-09-24
CN112004925B (zh) 2024-09-27
CN113795574A (zh) 2021-12-14
CA3093347A1 (fr) 2019-10-10
EP3947653A1 (fr) 2022-02-09
IL277740A (en) 2020-11-30
TN2020000187A1 (en) 2022-04-04
JP2021520203A (ja) 2021-08-19
US20240317814A1 (en) 2024-09-26
CN112004925A (zh) 2020-11-27
WO2019193119A1 (fr) 2019-10-10
US20220228167A1 (en) 2022-07-21
BR112020020223A2 (pt) 2021-01-19
JP2022526405A (ja) 2022-05-24
EP3775173A1 (fr) 2021-02-17
US20210107948A1 (en) 2021-04-15
MA51353A1 (fr) 2021-09-30
NZ767516A (en) 2024-11-29
BR112021019821A2 (pt) 2021-12-07
RU2020136195A (ru) 2022-05-06
KR20210153047A (ko) 2021-12-16
IL286926A (en) 2021-10-31
JP7374119B2 (ja) 2023-11-06
AU2019439673A1 (en) 2021-10-07
ZA202005422B (en) 2023-03-29
AU2019249890B2 (en) 2024-10-10
US12037362B2 (en) 2024-07-16

Similar Documents

Publication Publication Date Title
MA51353B1 (fr) Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit
PH12018502387A1 (en) Adeno-associated virus variant capsids and methods of use thereof
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
PH12020550706A1 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
PH12020551938A1 (en) Liver targeting adeno-associated viral vectors
MX2021011468A (es) Vectores de virus adenoasociados recombinantes.
MA49514B1 (fr) Particules virales recombinantes avec tropisme modifié, et leurs utilisations pour l'introduction ciblée de matériel génétique dans des cellules humaines
EA202092362A1 (ru) Вирусные векторы для нацеливания на ткани глаза
MX2020010466A (es) Vectores de virus que evitan anticuerpos.
EA201991911A1 (ru) Способы и композиции для переноса генов по сосудистой сети
BR112023024375A2 (pt) Vírus adeno-associado recombinante tendo capsídeo variante e sua aplicação
EP4461814A3 (fr) Génération de pah humain amélioré pour le traitement de pku sévère par thérapie de remplacement génique dirigée par le foie
MX2020010464A (es) Vectores de virus que evitan anticuerpos.
ZA202105975B (en) Variant aav capsids for intravitreal delivery
MX2022009982A (es) Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5.
WO2021030225A3 (fr) Variants de capsides aav pour le ciblage de cellules de glioblastome humain
PH12022551878A1 (en) Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
MA39439A1 (fr) Vecteurs aav pour thérapie génique de la rétine et du snc
MY186651A (en) Adeno-associated virus variant capsids and methods of use thereof
MA64619A1 (fr) Protéine modifiée séparée vp1 de capside aav5
EA202092363A1 (ru) Уклоняющиеся от антител вирусные векторы
MA40818B2 (fr) Administration améliorée de particules virales au striatum et au cortex
NZ749467A (en) Novel adeno-associated virus capsid proteins