MX2018007230A - Terapia genica para trastornos oculares. - Google Patents

Terapia genica para trastornos oculares.

Info

Publication number
MX2018007230A
MX2018007230A MX2018007230A MX2018007230A MX2018007230A MX 2018007230 A MX2018007230 A MX 2018007230A MX 2018007230 A MX2018007230 A MX 2018007230A MX 2018007230 A MX2018007230 A MX 2018007230A MX 2018007230 A MX2018007230 A MX 2018007230A
Authority
MX
Mexico
Prior art keywords
ocular disorders
adeno
nucleic acid
acid molecule
sequence encoding
Prior art date
Application number
MX2018007230A
Other languages
English (en)
Inventor
Sun Junwei
Bennett Jean
Bennicelli Jeannette
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 MX2018007230A publication Critical patent/MX2018007230A/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • C07K14/46—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
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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, un gato, un perro, una oveja o un primate no humano.
MX2018007230A 2015-12-14 2016-12-13 Terapia genica para trastornos oculares. MX2018007230A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562266789P 2015-12-14 2015-12-14
PCT/US2016/066402 WO2017106202A2 (en) 2015-12-14 2016-12-13 Gene therapy for ocular disorders

Publications (1)

Publication Number Publication Date
MX2018007230A true MX2018007230A (es) 2018-11-09

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 After (1)

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

Country Status (13)

Country Link
US (2) US11090392B2 (es)
EP (2) EP3795180A1 (es)
JP (2) JP7057281B2 (es)
KR (1) KR20180099719A (es)
CN (1) CN109069668B (es)
AU (1) AU2016370487C1 (es)
BR (1) BR112018011838A2 (es)
CA (1) CA3008264A1 (es)
ES (1) ES2826384T3 (es)
HK (1) HK1258518A1 (es)
MX (2) MX2018007230A (es)
RU (1) RU2762747C2 (es)
WO (1) WO2017106202A2 (es)

Families Citing this family (9)

* 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
PL3377637T3 (pl) * 2016-04-08 2020-09-07 Krystal Biotech, Inc. Kompozycje do stosowania w sposobach leczenia ran, zaburzeń i chorób skóry
EP3490613A4 (en) * 2016-07-26 2020-03-04 Cornell University GENE THERAPY FOR THE TREATMENT OF ALDEHYDE DEHYDROGENASE DEFICIT
EP3638316A4 (en) * 2017-06-14 2021-03-24 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR EYE DISORDERS
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
US20240091380A1 (en) 2021-02-01 2024-03-21 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
BR112023016997A2 (pt) * 2021-02-26 2023-11-07 Logicbio Therapeutics Inc Fabricação e uso de vetores aav recombinantes
BR112023020209A2 (pt) 2021-04-02 2023-12-19 Krystal Biotech Inc Genoma do vírus do herpes recombinante, vírus do herpes, composição farmacêutica, uso do vírus do herpes ou da composição farmacêutica, método para expressar, realçar, aumentar, ampliar e/ou suplementar os níveis de um polipeptídeo imunomodulatório, método para prover alívio profilático, paliativo ou terapêutico, e, método para tratar câncer
WO2023014892A1 (en) * 2021-08-04 2023-02-09 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
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869305A (en) 1992-12-04 1999-02-09 The 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
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
AU781958C (en) 1999-08-09 2006-03-30 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
DE60139471D1 (de) 2000-06-01 2009-09-17 Univ North Carolina Verfahren und zusammensetzungen zur kontrollierter abgabe von rekombinant parvovirus vektoren
JP2005500008A (ja) 2001-01-19 2005-01-06 インサイト・ゲノミックス・インコーポレイテッド 受容体および膜結合タンパク質
EP1381276A4 (en) 2001-04-13 2005-02-02 Univ Pennsylvania METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida 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
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 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
US20110305772A1 (en) 2009-02-26 2011-12-15 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
WO2011034947A2 (en) * 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
CA2793633A1 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP3591055A1 (en) 2011-01-07 2020-01-08 Applied Genetic Technologies Corporation Promoters derived from the 5' nontranslated region (5'-ntr) of the human cyclic nucleotide gated beta 3 (cngb3) gene, expression cassettes and vectors comprising said promoters, for use treating achromatopsia and other diseases
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and 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
WO2013063601A1 (en) * 2011-10-28 2013-05-02 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
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
WO2014011210A1 (en) 2012-07-11 2014-01-16 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
WO2014024282A1 (ja) 2012-08-08 2014-02-13 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
WO2014124282A1 (en) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
ES2704677T3 (es) 2013-04-18 2019-03-19 Fond Telethon Suministro eficaz de genes grandes por vectores de AAV duales
CA2912525A1 (en) 2013-05-16 2014-11-20 Applied Genetic Technologies Corporation Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
CA2942776C (en) 2014-03-17 2023-01-24 Adverum Biotechnologies, Inc. Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter
AU2015247777B2 (en) * 2014-04-15 2019-03-21 Beacon Therapeutics Limited 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
BR112018011838A2 (pt) 2018-12-04
US20200061209A1 (en) 2020-02-27
JP2019502378A (ja) 2019-01-31
EP3795180A1 (en) 2021-03-24
RU2018125468A (ru) 2020-01-16
AU2016370487A1 (en) 2018-07-05
WO2017106202A3 (en) 2017-08-31
US11090392B2 (en) 2021-08-17
JP7057281B2 (ja) 2022-04-19
MX2022010959A (es) 2022-10-07
CN109069668A (zh) 2018-12-21
US20210330816A1 (en) 2021-10-28
CA3008264A1 (en) 2017-06-22
RU2018125468A3 (es) 2020-10-22
CN109069668B (zh) 2023-04-18
AU2016370487C1 (en) 2022-03-03
EP3389724B1 (en) 2020-07-01
JP2022088656A (ja) 2022-06-14
KR20180099719A (ko) 2018-09-05
ES2826384T3 (es) 2021-05-18
WO2017106202A2 (en) 2017-06-22
AU2016370487B2 (en) 2021-08-05
EP3389724A2 (en) 2018-10-24
HK1258518A1 (zh) 2019-11-15
RU2762747C2 (ru) 2021-12-22

Similar Documents

Publication Publication Date Title
MX2022010959A (es) Terapia genica para trastornos oculares.
IL252917A0 (en) Vectors and vectors for gene therapy for use in the treatment of Wilson's disease
AU2024202345A1 (en) Nucleic acid carriers and therapeutic methods of use
MX2017007127A (es) Composiciones que comprenden polipeptidos que tienen actividad xilanasa y polipeptidos que tienen actividad arabinofuranosidasa.
IL280652A (en) Nucleic acid molecules and their use for non-viral gene therapy
SI3233129T1 (sl) Konstrukti nukleinske kisline in vektorji genske terapije za uporabo pri zdravljenju Wilsonove bolezni in drugih stanj
IL278058A (en) Oligonucleotide compounds of 5-cap-tetranucleotide or higher and their uses in RNA production, protein expression and medicine
PL3159409T3 (pl) Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a
WO2014179331A3 (en) Devices, systems and methods for optogenetic modulation of action potentials in target cells
MX2016013832A (es) Composiciones y metodos para tratar hemoglobinopatias.
PH12018500568A1 (en) Antisense nucleic acid
CY1123793T1 (el) Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθεια
GB2571603B (en) Hair treatment agent with anti-dandruff action
GB201513098D0 (en) Catalytically active protein aggregates and methods for producing the same
MX2018013875A (es) Composiciones que comprenden polipeptidos que tienen actividad galactanasa y polipeptidos que tienen actividad beta-galactosidasa.
EP3720509A4 (en) GLOBING THERAPY FOR THE TREATMENT OF HEMOGLOBINOPATHIES
PT3253781T (pt) Proteína dkk2 modificada, ácido nucleico que codifica a mesma, método de preparação da mesma e uso da mesma
IL274363B1 (en) Use of a specific anti-protein siRNA for the treatment of hemophilia
Keswani et al. Diversity, population and habitat used by spiders in bananaagro-ecosystem
MX2018014434A (es) Composiciones que comprenden polipeptidos que tienen actividad galactanasa y polipeptidos que tienen actividad beta-galactosidasa.
EP3372249C0 (en) AAV/UPR-PLUS VIRUS, UPR-PLUS FUSION PROTEIN, GENETIC TREATMENT METHOD AND USE THEREOF IN THE TREATMENT OF NEURODEGENERATIVE DISEASES SUCH AS PARKINSON'S DISEASE AND HUNTINGTON'S DISEASE
GB201522416D0 (en) Wilson's disease gene therapy
EP3677680A4 (en) SMALL NUCLEIC ACID ANTISENS GUIDE AND CORRESPONDING USE
EP3660163A4 (en) NUCLEIC ACID HOMOGENIZATION METHOD AND KIT AND USES THEREOF
MX2021007688A (es) Metodos para formar poliplejos.