MX2022010959A - Terapia genica para trastornos oculares. - Google Patents

Terapia genica para trastornos oculares.

Info

Publication number
MX2022010959A
MX2022010959A MX2022010959A MX2022010959A MX2022010959A MX 2022010959 A MX2022010959 A MX 2022010959A MX 2022010959 A MX2022010959 A MX 2022010959A MX 2022010959 A MX2022010959 A MX 2022010959A MX 2022010959 A MX2022010959 A MX 2022010959A
Authority
MX
Mexico
Prior art keywords
ocular disorders
adeno
nucleic acid
acid molecule
sequence encoding
Prior art date
Application number
MX2022010959A
Other languages
English (en)
Inventor
Junwei Sun
Jean Bennett
Jeannette Bennicelli
Original Assignee
Univ Pennsylvania
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 Pennsylvania filed Critical Univ Pennsylvania
Publication of MX2022010959A publication Critical patent/MX2022010959A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • 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
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/04—Uses of viruses as vector in vivo
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/002—Vectors comprising a special translation-regulating system controllable or inducible
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

Landscapes

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

Abstract

Se proporcionan composiciones y métodos para tratar trastornos oculares en un sujeto; en un aspecto se proporciona un vector viral adenoasociado que incluye una molécula de ácido nucleico que comprende una secuencia que codifica CNGA3; en otro aspecto, se proporciona un vector viral adenoasociado que incluye una molécula de ácido nucleico que comprende una secuencia que codifica CNGB3; en otro aspecto se proporciona un vector viral adenoasociado que incluye una molécula de ácido nucleico que comprende una secuencia que codifica REP-1; en modalidades deseadas, el sujeto es un ser humano, gato, perro, oveja o primate no humano.
MX2022010959A 2015-12-14 2018-06-13 Terapia genica para trastornos oculares. MX2022010959A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562266789P 2015-12-14 2015-12-14

Publications (1)

Publication Number Publication Date
MX2022010959A true MX2022010959A (es) 2022-10-07

Family

ID=58455636

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018007230A MX2018007230A (es) 2015-12-14 2016-12-13 Terapia genica para trastornos oculares.
MX2022010959A MX2022010959A (es) 2015-12-14 2018-06-13 Terapia genica para trastornos oculares.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2018007230A MX2018007230A (es) 2015-12-14 2016-12-13 Terapia genica para trastornos oculares.

Country Status (11)

Country Link
US (2) US11090392B2 (es)
EP (2) EP3795180B1 (es)
JP (2) JP7057281B2 (es)
KR (1) KR20180099719A (es)
CN (1) CN109069668B (es)
AU (1) AU2016370487C1 (es)
CA (1) CA3008264A1 (es)
ES (2) ES3061210T3 (es)
MX (2) MX2018007230A (es)
RU (1) RU2762747C2 (es)
WO (1) WO2017106202A2 (es)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017106202A2 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
SI3377637T1 (sl) * 2016-04-08 2020-08-31 Krystal Biotech, Inc. Sestavki za uporabo pri postopkih za zdravljenje ran, motenj in bolezni kože
KR102621134B1 (ko) * 2016-07-26 2024-01-04 코넬 유니버시티 알데히드 데히드로게나제 결핍의 치료를 위한 유전자 요법
US11827898B2 (en) 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
CN112041434A (zh) 2018-04-27 2020-12-04 克里斯托生物技术股份有限公司 用于美容性应用的编码一种或多种美容蛋白的重组核酸
MX2021008832A (es) 2019-02-08 2021-09-08 Krystal Biotech Inc Composiciones y metodos para la administracion de polipeptidos cftr.
JP7637060B2 (ja) * 2019-03-04 2025-02-27 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 Ａｋｔ経路を標的とする神経保護遺伝子療法
CN110889859A (zh) * 2019-11-11 2020-03-17 珠海上工医信科技有限公司 一种用于眼底图像血管分割的u型网络
EP4150051A4 (en) * 2020-04-27 2025-05-21 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF CDKL5 DEFICIENCY DISORDER (CDD)
US11357870B2 (en) * 2020-09-02 2022-06-14 4D Molecular Therapeutics Inc. Codon optimized REP1 genes and uses thereof
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022182986A1 (en) 2021-02-26 2022-09-01 Logicbio Therapeutics, Inc. Manufacturing and use of recombinant aav vectors
AU2022246917A1 (en) 2021-04-02 2023-10-12 Krystal Biotech, Inc. Viral vectors for cancer therapy
US20240335562A1 (en) * 2021-08-04 2024-10-10 Alpine Biotherapeutics Corporation Sustainable ocular cell-mediated intraocular delivery of cellular therapeutics for treatment of ocular diseases or disorders

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
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
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
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
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
EP2369002A1 (en) 1999-08-09 2011-09-28 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
CA2410901C (en) 2000-06-01 2012-03-20 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
EP1409677A2 (en) 2001-01-19 2004-04-21 Incyte Genomics, Inc. Receptors and membrane-associated proteins
WO2002082904A2 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
EP3085389A1 (en) 2005-04-07 2016-10-26 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
WO2006132118A1 (ja) 2005-06-09 2006-12-14 Matsushita Electric Industrial Co., Ltd. 振幅誤差補償装置及び直交度誤差補償装置
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
WO2010005533A2 (en) 2008-06-30 2010-01-14 The Johns Hopkins University Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa
WO2010099383A2 (en) 2009-02-26 2010-09-02 The Johns Hopkins University Compositions and methods for ex vivo hepatic nucleic acid delivery
RU2444977C2 (ru) * 2009-08-03 2012-03-20 Федеральное государственное учреждение "МОСКОВСКИЙ НАУЧНО-ИССЛЕДОВАТЕЛЬСКИЙ ИНСТИТУТ ГЛАЗНЫХ БОЛЕЗНЕЙ ИМЕНИ ГЕЛЬМГОЛЬЦА ФЕДЕРАЛЬНОГО АГЕНТСТВА ПО ВЫСОКОТЕХНОЛОГИЧНОЙ МЕДИЦИНСКОЙ ПОМОЩИ" Способ диагностики ахроматопсии
US20120172419A1 (en) * 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
KR20130040844A (ko) 2010-03-29 2013-04-24 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 약학적으로 유발된 전이유전자 제거 시스템
WO2012094560A2 (en) * 2011-01-07 2012-07-12 Applied Genetic Technologies Corporation Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
WO2012158757A1 (en) 2011-05-16 2012-11-22 The Trustees Of The University Of Pennsylvania Proviral plasmids for production of recombinant adeno-associated virus
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
AU2012327236B2 (en) 2011-10-28 2016-11-10 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
WO2013086515A1 (en) 2011-12-09 2013-06-13 The Johns Hopkins University Compositions and methods for the prevention or treatment of diabetic complications
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
EP2872183B1 (en) 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
JP6129180B2 (ja) 2012-08-08 2017-05-17 日本パーカライジング株式会社 金属表面処理液、金属基材の表面処理方法及びそれにより得られた金属基材
ES2741502T3 (es) 2013-02-08 2020-02-11 Univ Pennsylvania Cápside de AAV8 modificada para la transferencia génica para terapias de retina
EP2986635B1 (en) 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
CN112779255A (zh) 2013-05-16 2021-05-11 应用遗传科技公司 用于治疗色盲和其它疾病的启动子、表达盒、载体、药盒和方法
GB201322798D0 (en) * 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
US9534225B2 (en) * 2014-04-15 2017-01-03 Applied Genetic Technologies Corporation Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (RPGR)
US20160206704A1 (en) * 2015-01-16 2016-07-21 Isis Innovation Limited Method
WO2017106202A2 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders

Also Published As

Publication number Publication date
AU2016370487C1 (en) 2022-03-03
JP2019502378A (ja) 2019-01-31
US20210330816A1 (en) 2021-10-28
RU2018125468A3 (es) 2020-10-22
CN109069668A (zh) 2018-12-21
BR112018011838A2 (pt) 2018-12-04
JP2022088656A (ja) 2022-06-14
JP7057281B2 (ja) 2022-04-19
ES3061210T3 (en) 2026-03-31
US20200061209A1 (en) 2020-02-27
CA3008264A1 (en) 2017-06-22
CN109069668B (zh) 2023-04-18
WO2017106202A2 (en) 2017-06-22
RU2018125468A (ru) 2020-01-16
US11090392B2 (en) 2021-08-17
HK1258518A1 (zh) 2019-11-15
EP3795180B1 (en) 2025-11-05
EP3389724A2 (en) 2018-10-24
EP3389724B1 (en) 2020-07-01
AU2016370487A1 (en) 2018-07-05
EP3795180A1 (en) 2021-03-24
US12403204B2 (en) 2025-09-02
RU2762747C2 (ru) 2021-12-22
KR20180099719A (ko) 2018-09-05
MX2018007230A (es) 2018-11-09
WO2017106202A3 (en) 2017-08-31
AU2016370487B2 (en) 2021-08-05
ES2826384T3 (es) 2021-05-18

Similar Documents

Publication Publication Date Title
WO2017106202A3 (en) Gene therapy for ocular disorders
IL280652A (en) Nucleic acid molecules and their use for non-viral gene therapy
MX383142B (es) Composiciones que comprenden polipéptidos que tienen actividad xilanasa y polipéptidos que tienen actividad arabinofuranosidasa.
IL252917A0 (en) Vectors and vectors for gene therapy for use in the treatment of Wilson's disease
PT3233129T (pt) Construções de ácido nucleico e vetores de terapia génica para utilização no tratamento de doença de wilson e outras condições
IL271389A (en) Targeted non-viral dna insertions
EP3440205A4 (en) NONINVASIVE DIAGNOSIS BY SEQUENCING OF 5-HYDROXYMETHYLATED CELL-FREE DNA
EP3741849A3 (en) Protease variants and polynucleotides encoding same
WO2014179331A3 (en) Devices, systems and methods for optogenetic modulation of action potentials in target cells
HUE047502T2 (hu) Antiszensz nukleinsav Duchenne izomsorvadás kezelésében történõ alkalmazásra
HK1256909A1 (zh) Glp-1和其在用於治疗代谢疾病的组合物中的用途
MX2016013832A (es) Composiciones y metodos para tratar hemoglobinopatias.
PH12018500568B1 (en) Antisense nucleic acid
AU2022271491B2 (en) Antisense oligonucleotides for use in treating alzheimer's disease
CY1123793T1 (el) Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθεια
IL291922B2 (en) FAP-activated medical materials and related uses
GB2571603B (en) Hair treatment agent with anti-dandruff action
HK1257024A1 (zh) 用於治疗伴侣动物的aav-epo
MX2018013875A (es) Composiciones que comprenden polipeptidos que tienen actividad galactanasa y polipeptidos que tienen actividad beta-galactosidasa.
PT3253781T (pt) Proteína dkk2 modificada, ácido nucleico que codifica a mesma, método de preparação da mesma e uso da mesma
GB201522416D0 (en) Wilson's disease gene therapy
IL274363B1 (en) Use of a specific anti-protein siRNA for the treatment of hemophilia
MX2018014434A (es) Composiciones que comprenden polipeptidos que tienen actividad galactanasa y polipeptidos que tienen actividad beta-galactosidasa.
王剑 FE 70-200mm F4 G OSS
Appannacharya Sri Jagannatadasara Kritigalu-K Appannacharya