[go: up one dir, main page]

CO6640236A2 - Compisicones de ciclasa de maav guanulato y métodos para tratar la amaurosis congénita de leber 1(lca1) - Google Patents

Compisicones de ciclasa de maav guanulato y métodos para tratar la amaurosis congénita de leber 1(lca1)

Info

Publication number
CO6640236A2
CO6640236A2 CO12210917A CO12210917A CO6640236A2 CO 6640236 A2 CO6640236 A2 CO 6640236A2 CO 12210917 A CO12210917 A CO 12210917A CO 12210917 A CO12210917 A CO 12210917A CO 6640236 A2 CO6640236 A2 CO 6640236A2
Authority
CO
Colombia
Prior art keywords
methods
leber
congenital amaurosis
cyclase
guanylate cyclase
Prior art date
Application number
CO12210917A
Other languages
English (en)
Inventor
Shannon Elizabeth Boye
William W Hauswirth
Sanford Leon Boye
Original Assignee
Univ Florida
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 Florida filed Critical Univ Florida
Publication of CO6640236A2 publication Critical patent/CO6640236A2/es

Links

Classifications

    • 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
    • C12N15/861—Adenoviral 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51—Lyases (4)
    • 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
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • 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
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y406/00—Phosphorus-oxygen lyases (4.6)
    • C12Y406/01—Phosphorus-oxygen lyases (4.6.1)
    • C12Y406/01002—Guanylate cyclase (4.6.1.2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

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

Abstract

Se revelan composiciones de vectores virales que comprenden secuencias de polinucleótidos que expresan una o varias proteínas de guanilato ciclasa de mamíferos biológicamente activas. También se revelan métodos para su uso en la prevención, el tratamiento y/o la mejora de al menos uno o varios síntomas de una enfermedad, trastorno, condición anormal o disfunción resultante al menos en parte de una deficiencia de guanilato ciclasa in vivo. En formas de realización particulares, el uso de vectores virales adenoasociados recombiriantes (rAAV) para tratar o mejorar síntomas de amaurosis congénita de Leber, así como otras condiciones causadas por ausencia o reducción en la expresión de una guanilato ciclasa 1 específica de la retina funcional (retGCl).
CO12210917A 2010-04-23 2012-11-21 Compisicones de ciclasa de maav guanulato y métodos para tratar la amaurosis congénita de leber 1(lca1) CO6640236A2 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US32752110P 2010-04-23 2010-04-23

Publications (1)

Publication Number Publication Date
CO6640236A2 true CO6640236A2 (es) 2013-03-22

Family

ID=44533055

Family Applications (1)

Application Number Title Priority Date Filing Date
CO12210917A CO6640236A2 (es) 2010-04-23 2012-11-21 Compisicones de ciclasa de maav guanulato y métodos para tratar la amaurosis congénita de leber 1(lca1)

Country Status (28)

Country Link
US (3) US9816108B2 (es)
EP (3) EP2561067B1 (es)
JP (4) JP5897549B2 (es)
KR (3) KR101984145B1 (es)
CN (2) CN105821079B (es)
AU (1) AU2011242527B2 (es)
BR (1) BR112012026730A8 (es)
CA (1) CA2796399C (es)
CL (1) CL2012002969A1 (es)
CO (1) CO6640236A2 (es)
CY (1) CY1123008T1 (es)
DK (2) DK2561067T3 (es)
EA (2) EA202091105A1 (es)
ES (2) ES2711256T3 (es)
HR (1) HRP20190144T1 (es)
HU (1) HUE041571T2 (es)
IL (2) IL222651B (es)
LT (1) LT2561067T (es)
MX (1) MX351865B (es)
NZ (1) NZ602897A (es)
PL (2) PL3486320T3 (es)
PT (2) PT3486320T (es)
RS (1) RS58434B1 (es)
SG (4) SG184876A1 (es)
SI (1) SI2561067T1 (es)
SM (1) SMT201900097T1 (es)
TR (1) TR201901377T4 (es)
WO (1) WO2011133933A2 (es)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
BR112014031788A2 (pt) 2012-06-19 2017-06-27 Univ Florida composições e métodos para o tratamento de diabetes
FR3008618A1 (fr) * 2013-07-19 2015-01-23 Univ Paris Curie Utilisation de composes pour restaurer la reponse a la lumiere des cellules de la retine
HUE047996T2 (hu) 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
JP6827320B2 (ja) 2013-08-05 2021-02-10 アカデミシュ・ジークンホイス・ライデン・ハー・オー・デー・エン・ライドス・ユニヴェルシタイル・メディシュ・セントルム ＬＣＡ−８及び進行性ＲＰを治療するための組換えＡＡＶ−Ｃｒｕｍｂｓホモログ組成物及び方法
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CN108138159A (zh) 2015-06-23 2018-06-08 费城儿童医院 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途
KR20230167138A (ko) * 2017-05-05 2023-12-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
WO2019046341A2 (en) 2017-08-28 2019-03-07 University Of Florida Research Foundation, Incorporated METHODS AND COMPOSITIONS FOR TREATING RETINAL DYSTROPHY OF CONES AND STICKS
JP2021502978A (ja) 2017-11-15 2021-02-04 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法
US11845952B2 (en) 2018-02-07 2023-12-19 Nippon Medical School Foundation Adeno-associated virus vector
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2019210181A1 (en) 2018-04-27 2019-10-31 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
CN112384220A (zh) 2018-07-11 2021-02-19 塞科里昂医疗股份有限公司 sGC刺激剂治疗线粒体障碍的用途
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
WO2020154535A1 (en) * 2019-01-23 2020-07-30 University Of Florida Research Foundation, Incorporated Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
WO2020163761A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
CN114144518A (zh) 2019-02-15 2022-03-04 爱科霍拉有限公司 用于基因疗法的双亮氨酸拉链激酶抑制剂
JP2024016295A (ja) * 2020-11-30 2024-02-07 タカラバイオ株式会社 二価の陽イオンを利用した非エンベロープウイルスの製造方法
CN112852976B (zh) * 2021-03-17 2023-10-31 湖北省农业科学院畜牧兽医研究所 蛋鸡ncs1基因中与后期产蛋性状相关的分子标记及其应用
KR20240055728A (ko) * 2021-07-14 2024-04-29 메이라지티엑스 유케이 Ii 리미티드 Retgc 유전자 요법

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4237224A (en) 1974-11-04 1980-12-02 Board Of Trustees Of The Leland Stanford Jr. University Process for producing biologically functional molecular chimeras
US4554101A (en) 1981-01-09 1985-11-19 New York Blood Center, Inc. Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity
US4883750A (en) 1984-12-13 1989-11-28 Applied Biosystems, Inc. Detection of specific sequences in nucleic acids
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4800159A (en) 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
AU622104B2 (en) 1987-03-11 1992-04-02 Sangtec Molecular Diagnostics Ab Method of assaying of nucleic acids, a reagent combination and kit therefore
AU622426B2 (en) 1987-12-11 1992-04-09 Abbott Laboratories Assay using template-dependent nucleic acid probe reorganization
DE68908054T2 (de) 1988-01-21 1994-03-10 Genentech Inc Verstärkung und nachweis von nukleinsäuresequenzen.
CA1340807C (en) 1988-02-24 1999-11-02 Lawrence T. Malek Nucleic acid amplification process
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
CA2039718C (en) 1989-08-31 2003-02-25 John J. Rossi Chimeric dna-rna catalytic sequences
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
AU6505690A (en) 1989-09-26 1991-04-28 Richard R Behringer Erythroid-specific gene expression system
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー．ファウ． 修飾リボザイム
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5145684A (en) 1991-01-25 1992-09-08 Sterling Drug Inc. Surface modified drug nanoparticles
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
AU671093B2 (en) 1992-01-07 1996-08-15 Elan Pharmaceuticals, Inc. Transgenic animal models for alzheimer's disease
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
EP1251170A3 (en) 1992-07-17 2002-10-30 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of NF-kappaB dependent animal diseases
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5639940A (en) 1994-03-03 1997-06-17 Pharmaceutical Proteins Ltd. Production of fibrinogen in transgenic animals
US5789655A (en) 1994-05-13 1998-08-04 The Regents Of The University Of California Transgenic animals expressing artificial epitope-tagged proteins
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
US6225291B1 (en) 1997-04-21 2001-05-01 University Of Florida Rod opsin mRNA-specific ribozyme compositions and methods for the treatment of retinal diseases
CA2745131C (en) * 1998-05-28 2016-08-09 John A. Chiorini Aav5 vector and uses thereof
WO2002082904A2 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
CN1381579A (zh) * 2001-04-18 2002-11-27 上海博德基因开发有限公司 一种多肽——鸟苷酸环化酶－13.53和编码这种多肽的多核苷酸
US20030003582A1 (en) * 2001-05-08 2003-01-02 Tranzyme, Inc. Trans-viral vector mediated gene transfer to the retina
EP1583821A4 (en) * 2003-01-15 2007-07-18 Millennium Pharm Inc METHODS AND COMPOSITIONS FOR TREATING UROLOGICAL DISORDERS USING GENES 44390, 54181, 211, 5687, 884, 1405, 636, 4421, 5410, 30905, 2045, 16405, 18560, 2047, 33751, 52872, 14063, 20739, 32544, 43239, 44373, 51164, 53010, 16852, 1587, 2207, 22245, 2387, 52908, 69112, 14990, 18547, 115, 579
EP1616965A1 (en) * 2004-07-16 2006-01-18 AXXAM S.r.l. Methods and assays for detecting guanylate cyclase activity
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
WO2007078599A2 (en) 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
CA2694091A1 (en) * 2007-10-01 2009-04-09 Alcon Research, Ltd. Self complementary aav-mediated delivery of interfering rna molecules to treat or prevent ocular disorders
US8312249B1 (en) 2008-10-10 2012-11-13 Apple Inc. Dynamic trampoline and structured code generation in a signed code environment
FR2957821B1 (fr) 2010-03-24 2014-08-29 Inst Francais Du Petrole Nouvelle zone de regeneration du catalyseur divisee en secteurs pour unites catalytiques regeneratives

Also Published As

Publication number Publication date
KR20190060881A (ko) 2019-06-03
WO2011133933A3 (en) 2012-01-05
US20220186260A1 (en) 2022-06-16
EP2561067B1 (en) 2018-11-14
KR20130095643A (ko) 2013-08-28
KR101984145B1 (ko) 2019-05-30
RS58434B1 (sr) 2019-04-30
SG184876A1 (en) 2012-11-29
HUE041571T2 (hu) 2019-05-28
BR112012026730A2 (pt) 2015-09-22
CL2012002969A1 (es) 2013-05-31
AU2011242527B2 (en) 2016-05-19
HRP20190144T1 (hr) 2019-03-22
DK3486320T3 (da) 2022-04-11
MX351865B (es) 2017-10-30
PL3486320T3 (pl) 2022-05-09
KR20200047642A (ko) 2020-05-07
EA035893B1 (ru) 2020-08-27
EP3486320A1 (en) 2019-05-22
EP4056700A1 (en) 2022-09-14
TR201901377T4 (tr) 2019-02-21
JP2013526854A (ja) 2013-06-27
PL2561067T3 (pl) 2019-05-31
JP6533246B2 (ja) 2019-06-19
CN102918152A (zh) 2013-02-06
PT3486320T (pt) 2022-03-30
EA202091105A1 (ru) 2020-12-30
LT2561067T (lt) 2019-03-12
SG10201913135YA (en) 2020-02-27
CA2796399A1 (en) 2011-10-27
JP2019107037A (ja) 2019-07-04
CN102918152B (zh) 2016-04-13
JP2017114914A (ja) 2017-06-29
CN105821079A (zh) 2016-08-03
SI2561067T1 (sl) 2019-04-30
EP2561067A2 (en) 2013-02-27
US9816108B2 (en) 2017-11-14
PT2561067T (pt) 2019-02-04
SG10201502759TA (en) 2015-06-29
JP5897549B2 (ja) 2016-03-30
SMT201900097T1 (it) 2019-02-28
CN105821079B (zh) 2021-10-26
BR112012026730A8 (pt) 2018-07-03
IL264649B (en) 2021-01-31
WO2011133933A2 (en) 2011-10-27
CY1123008T1 (el) 2021-10-29
EP3486320B1 (en) 2022-01-19
US20130210895A1 (en) 2013-08-15
DK2561067T3 (en) 2019-03-11
MX2012012367A (es) 2012-11-16
IL222651B (en) 2019-02-28
ES2711256T3 (es) 2019-04-30
NZ602897A (en) 2014-09-26
EA201291103A1 (ru) 2013-10-30
CA2796399C (en) 2021-08-24
IL222651A0 (en) 2012-12-31
ES2911185T3 (es) 2022-05-18
JP2015091273A (ja) 2015-05-14
US20180100165A1 (en) 2018-04-12
SG10201800541SA (en) 2018-03-28

Similar Documents

Publication Publication Date Title
CO6640236A2 (es) Compisicones de ciclasa de maav guanulato y métodos para tratar la amaurosis congénita de leber 1(lca1)
EP2540825A3 (en) Cleaning compositions comprising amylase variants reference to a sequence listing
WO2016130589A3 (en) VARIANT RNAi
MY169328A (en) Compositions for the treatment of dry eye
BR112015000161A2 (pt) composições de vetor de vírus adeno-associado modificado
ECSP12011816A (es) Cepas de lactobacillus plantarum como agentes hipocolesterolémicos
BR112014009790A2 (pt) composto para modulação antisense da expressão de gccr, seu uso e composição
MX2014003289A (es) Preparacion y composiciones de azufre con valencia cero, altamente bio-disponibles, y sus usos.
BR112014000542A2 (pt) composições de peroxidase eosinofílica e métodos de uso das mesmas
WO2011042902A3 (en) Microcapsules comprising benzoyl peroxide and topical compositions comprising them
MX2013004062A (es) Analogos de ciclosporina.
EA202290001A1 (ru) Векторы aav с промотором миелинового белка ноль и их применение для лечения заболеваний, связанных со шванновскими клетками, таких как болезнь шарко-мари-тута
ECSP088578A (es) Enzimas para estrés inmunológico reducido
EA201071369A1 (ru) Композиции для лечения выпадения волос
CL2008003332A1 (es) Compuestos derivados de ciclopentano sustituido, como agentes hipotensores oculares; composicion oftalmologicamente aceptable; y uso para el tratamiento del glaucoma.
PH12015502100A1 (en) Reducing the risk of autoimmune disease
DOP2010000406A (es) Nifurtimox para el tratamiento de enfermedades causadas por trichomonadida
WO2015108856A3 (en) Methods for enhancing the delivery of active agents
ZA202003416B (en) Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
WO2012024419A3 (en) Bradykinin receptor agonists and uses thereof to treat ocular hypertension and glaucoma
NZ734845A (en) Use of short synthetic peptide for the treatment and/or prophylaxis of dry eye disease
WO2011073439A3 (en) Use of peptides as antidandruff agents
WO2010043972A3 (en) Bbeta (15-42) and its analogues combined with cyclosporine for treatment of reperfusion injury
WO2014144815A3 (en) Vectors and methods to treat ischemia
Tietjens Der Windkanal am Indian Institute of Science, Bangalore, Sud-Indien