RU2664673C2 - Векторы, кодирующие фактор жизнеспособности колбочек, полученный из палочек - Google Patents

Векторы, кодирующие фактор жизнеспособности колбочек, полученный из палочек Download PDF

Info

Publication number
RU2664673C2
RU2664673C2 RU2014121256A RU2014121256A RU2664673C2 RU 2664673 C2 RU2664673 C2 RU 2664673C2 RU 2014121256 A RU2014121256 A RU 2014121256A RU 2014121256 A RU2014121256 A RU 2014121256A RU 2664673 C2 RU2664673 C2 RU 2664673C2
Authority
RU
Russia
Prior art keywords
nucleic acid
rdcvf
cells
protein
vector
Prior art date
Application number
RU2014121256A
Other languages
English (en)
Russian (ru)
Other versions
RU2014121256A (ru
Inventor
Тианси ЛУО
Original Assignee
Веллстат Офтэлмикс Корпорэйшн
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 Веллстат Офтэлмикс Корпорэйшн filed Critical Веллстат Офтэлмикс Корпорэйшн
Publication of RU2014121256A publication Critical patent/RU2014121256A/ru
Application granted granted Critical
Publication of RU2664673C2 publication Critical patent/RU2664673C2/ru

Links

Images

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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K38/44—Oxidoreductases (1)
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • 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
    • A61P27/08—Mydriatics or cycloplegics
    • 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
    • A61P27/10—Ophthalmic agents for accommodation disorders, e.g. myopia
    • 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
    • A61P27/12—Ophthalmic agents for cataracts
    • 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
    • A61P27/14—Decongestants or antiallergics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • 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
    • C12N9/0004—Oxidoreductases (1.)
    • 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
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0051—Oxidoreductases (1.) acting on a sulfur group of donors (1.8)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • 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
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Psychiatry (AREA)
RU2014121256A 2011-10-27 2012-10-26 Векторы, кодирующие фактор жизнеспособности колбочек, полученный из палочек RU2664673C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161552155P 2011-10-27 2011-10-27
US61/552,155 2011-10-27
PCT/US2012/062106 WO2013063383A2 (en) 2011-10-27 2012-10-26 Vectors encoding rod-derived cone viability factor

Publications (2)

Publication Number Publication Date
RU2014121256A RU2014121256A (ru) 2015-12-10
RU2664673C2 true RU2664673C2 (ru) 2018-08-21

Family

ID=48168788

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2014121256A RU2664673C2 (ru) 2011-10-27 2012-10-26 Векторы, кодирующие фактор жизнеспособности колбочек, полученный из палочек

Country Status (14)

Country Link
US (2) US9265813B2 (enExample)
EP (1) EP2797613B1 (enExample)
JP (1) JP6293664B2 (enExample)
KR (1) KR102000141B1 (enExample)
CN (2) CN110777124A (enExample)
AU (1) AU2012321102C1 (enExample)
BR (1) BR112014010091B1 (enExample)
CA (1) CA2853379C (enExample)
DK (1) DK2797613T3 (enExample)
ES (1) ES2774779T3 (enExample)
MX (1) MX354516B (enExample)
PL (1) PL2797613T3 (enExample)
RU (1) RU2664673C2 (enExample)
WO (1) WO2013063383A2 (enExample)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2027889A1 (en) * 2007-06-05 2009-02-25 Institut National De La Sante Et De La Recherche Medicale (Inserm) New neuronal viability factor and use thereof
US10004780B2 (en) 2012-10-17 2018-06-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of age-related macular degeneration (AMD)
ES2744565T3 (es) 2014-04-25 2020-02-25 Genethon Tratamiento de la hiperbilirrubinemia
JP6684343B2 (ja) * 2015-05-21 2020-04-22 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ニューロン生存因子の相乗組み合わせ及びその使用
GB201516066D0 (en) 2015-09-10 2015-10-28 Young & Co Llp D Treatment of retinitis pigmentosa
AU2016370590B2 (en) 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
WO2017120294A1 (en) * 2016-01-05 2017-07-13 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of ocular disorders and blinding diseases
CN106148528B (zh) * 2016-07-11 2019-10-01 赵晨 一种遗传性Usher综合征的致病突变及其检测试剂
KR20260085174A (ko) * 2016-10-11 2026-06-08 파마 싱크, 엘엘씨 짧은 형태의 간상체 유래 원추체 생존능 인자와 친수성 펩타이드 사이의 융합 단백질
AU2018350700B2 (en) * 2017-10-16 2025-04-24 VeonGen Therapeutics GmbH AAV vectors
WO2019122403A1 (en) * 2017-12-22 2019-06-27 Sparingvision Constructs comprising neuronal viability factors and uses thereof
WO2019149648A1 (en) * 2018-01-30 2019-08-08 Universiteit Gent Compositions for use to treat cataract
CN108504685A (zh) * 2018-03-27 2018-09-07 宜明细胞生物科技有限公司 一种利用CRISPR/Cas9系统同源重组修复IL-2RG缺陷基因的方法
KR20250058061A (ko) * 2018-09-07 2025-04-29 유니버시티 오브 써던 캘리포니아 세포 시딩된 기질로부터의 치료적 상층액 및 관련 방법
KR20260086827A (ko) * 2018-11-14 2026-06-10 리젠엑스바이오 인크. 신경원성 세로이드 리포푸신증에 대한 유전자 요법
JP2022514863A (ja) * 2018-12-19 2022-02-16 ヴェルサメブ アーゲー タンパク質をコードするｒｎａ
CN114651003A (zh) 2019-09-10 2022-06-21 黑曜石疗法公司 用于可调调节的ca2-il15融合蛋白
JP7495756B2 (ja) * 2019-12-09 2024-06-05 北京中因科技有限公司 医薬の製造におけるＣＹＰ４Ｖ２およびＲｄＣＶＦの使用
CN111733174B (zh) * 2020-08-07 2021-02-09 北京大学第三医院（北京大学第三临床医学院） 一种分离的核酸分子及其用途
IL300738A (en) * 2020-08-28 2023-04-01 Biocad Joint Stock Co A vaccine for SARS-COV-2 based on AAV5
CN121285568A (zh) * 2023-03-30 2026-01-06 制药五有限责任公司 编码视杆细胞源性视锥细胞活力因子和人IgK信号序列的载体
WO2026072752A1 (en) 2024-09-27 2026-04-02 Pharma Cinq, Llc Rod-derived cone viability factor fusion protein

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050191747A1 (en) * 2002-02-04 2005-09-01 Tianci Luo Recombinant bovine immunodeficiency virus based gene transfer system
RU2345780C2 (ru) * 2003-05-02 2009-02-10 Дзе Скриппс Рисерч Инститьют Гематопоэтические стволовые клетки и способы лечения неоваскулярных заболеваний глаз с их помощью
US20110034546A1 (en) * 2008-04-15 2011-02-10 Genzyme Corporation Methods to produce rod-derived cone viability factor (rdcvf)

Family Cites Families (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5840293A (en) 1988-11-16 1998-11-24 Advanced Polymer Systems, Inc. Ionic beads for controlled release and adsorption
US5225347A (en) 1989-09-25 1993-07-06 Innovir Laboratories, Inc. Therapeutic ribozyme compositions and expression vectors
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
SG47470A1 (en) 1991-04-25 1998-04-17 Univ Brown Res Found Implantable biocompatible immunoisolatory vehicle for delivery of a selected therapeutic products
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US6040172A (en) 1992-08-14 2000-03-21 The Rockefeller University Defective DNA viral vector comprising a neural tissue-specific promoter for in vivo expression of a gene
KR950703640A (ko) 1992-10-09 1995-09-20 가일 케이,나우톤. 간 보존 세포(liver reserve cells)
GB9223084D0 (en) 1992-11-04 1992-12-16 Imp Cancer Res Tech Compounds to target cells
US5908623A (en) 1993-08-12 1999-06-01 Cytotherapeutics, Inc. Compositions and methods for the delivery of biologically active molecules using genetically altered cells contained in biocompatible immunoisolatory capsules
US5552311A (en) 1993-09-14 1996-09-03 University Of Alabama At Birmingham Research Foundation Purine nucleoside phosphorylase gene therapy for human malignancy
TW442569B (en) 1993-10-25 2001-06-23 Canji Inc Recombinant adenoviral vector
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5763416A (en) 1994-02-18 1998-06-09 The Regent Of The University Of Michigan Gene transfer into bone cells and tissues
US5942496A (en) 1994-02-18 1999-08-24 The Regent Of The University Of Michigan Methods and compositions for multiple gene transfer into bone cells
FR2716459B1 (fr) 1994-02-22 1996-05-10 Univ Paris Curie Système hôte-vecteur utilisable en thérapie génique.
EP0804249A2 (en) 1994-03-15 1997-11-05 Brown University Research Foundation Polymeric gene delivery system
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
EP0784690B1 (en) 1994-06-10 2006-08-16 Genvec, Inc. Complementary adenoviral vector systems and cell lines
CA2203809C (en) 1994-10-28 2008-06-03 James M. Wilson Recombinant adenovirus and methods of use thereof
US5843742A (en) 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
US5880102A (en) 1995-01-17 1999-03-09 Duke University Adenoviral vector system
US5707618A (en) 1995-03-24 1998-01-13 Genzyme Corporation Adenovirus vectors for gene therapy
DE19514541C2 (de) 1995-04-20 1998-01-15 Daimler Benz Ag Gaspedal mit Reibkörper
US5773289A (en) 1995-06-06 1998-06-30 University Of Pittsburgh AAV directed targeted integration
US5869040A (en) 1995-06-07 1999-02-09 Biogen, Inc Gene therapy methods and compositions
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
DE19541450C2 (de) 1995-11-07 1997-10-02 Gsf Forschungszentrum Umwelt Genkonstrukt und dessen Verwendung
US5830727A (en) 1995-11-18 1998-11-03 Human Gene Therapy Research Institute Herpes simplex virus amplicon mini-vector gene transfer system
US20030040467A1 (en) 1998-06-15 2003-02-27 Mary Ann Pelleymounter Ig/ob fusions and uses thereof.
US6299895B1 (en) 1997-03-24 2001-10-09 Neurotech S.A. Device and method for treating ophthalmic diseases
US6878544B2 (en) 1996-04-19 2005-04-12 Neurotech Sa Retinal cell lines with extended life-span and their applications
US6054142A (en) 1996-08-01 2000-04-25 Cyto Therapeutics, Inc. Biocompatible devices with foam scaffolds
US5994132A (en) 1996-10-23 1999-11-30 University Of Michigan Adenovirus vectors
CA2274314C (en) 1996-12-02 2007-03-13 Genemedicine, Inc. Insulin-like growth factor i (igf-i) expression system and methods of use
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6303136B1 (en) 1998-04-13 2001-10-16 Neurotech S.A. Cells or tissue attached to a non-degradable filamentous matrix encapsulated by a semi-permeable membrane
US5981225A (en) 1998-04-16 1999-11-09 Baylor College Of Medicine Gene transfer vector, recombinant adenovirus particles containing the same, method for producing the same and method of use of the same
US5994134A (en) 1998-05-04 1999-11-30 Canji, Inc. Viral production process
EP0974660A1 (en) 1998-06-19 2000-01-26 Stichting Instituut voor Dierhouderij en Diergezondheid (ID-DLO) Newcastle disease virus infectious clones, vaccines and diagnostic assays
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
US6146642A (en) 1998-09-14 2000-11-14 Mount Sinai School Of Medicine, Of The City University Of New York Recombinant new castle disease virus RNA expression systems and vaccines
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6303362B1 (en) 1998-11-19 2001-10-16 The Board Of Trustees Of The Leland Stanford Junior University Adenoviral vector and methods for making and using the same
US6361771B1 (en) 1999-04-06 2002-03-26 Neurotech S.A. ARPE-19 as a platform cell line for encapsulated cell-based delivery
US6864085B2 (en) 1999-12-14 2005-03-08 Novartis Ag Bovine immunodeficiency virus (BIV) based vectors
US7098030B2 (en) 1999-12-31 2006-08-29 Mirus Bio Corporation Polyampholytes for delivering polyions to a cell
IL140844A0 (en) 2001-01-10 2002-02-10 Polygene Ltd Cationic polysaccharide compositions
FR2823221B1 (fr) * 2001-04-06 2004-04-02 Univ Pasteur Sequences associees a la degenerescence retinienne et applications
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
CN1307300C (zh) 2002-07-25 2007-03-28 斯克里普斯研究学院 造血干细胞和治疗新生血管性眼部疾病的方法
DE10260805A1 (de) 2002-12-23 2004-07-22 Geneart Gmbh Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins
FR2870241B1 (fr) 2004-05-13 2015-02-27 Novartis Ag Facteur de viabilite des cones derive des batonnets ou rdcvf et applications
EP1971288B1 (en) 2005-12-30 2013-02-13 Neurotech USA, Inc. Micronized device for the delivery of biologically active molecules and methods of use thereof
MX2009009200A (es) 2007-03-01 2009-10-28 Advanced Vision Therapies Inc Tratamiento de enfermedades caracterizadas por la inflamacion.
EP2027889A1 (en) * 2007-06-05 2009-02-25 Institut National De La Sante Et De La Recherche Medicale (Inserm) New neuronal viability factor and use thereof
JP5710265B2 (ja) * 2007-12-06 2015-04-30 アンセルム（アンスチチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル） 網膜変性疾患の治療のための栄養因子
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
JP5748223B2 (ja) 2008-09-10 2015-07-15 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ニューロン生存因子およびその使用
US20120088819A1 (en) * 2008-10-31 2012-04-12 Makoto Inoue Method for enhancing expression of recombinant protein
DE102009016934A1 (de) 2009-04-08 2010-10-14 Daimler Ag Brennstoffzelle, Brennstoffzellenstapel und Verfahren zum Abdichten einer Brennstoffzelle
EP2486128A4 (en) 2009-10-08 2013-10-09 Neurotech Usa Inc USE OF THE PEDF IN AN ENCAPSULATED CELL DELIVERY SYSTEM

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050191747A1 (en) * 2002-02-04 2005-09-01 Tianci Luo Recombinant bovine immunodeficiency virus based gene transfer system
RU2345780C2 (ru) * 2003-05-02 2009-02-10 Дзе Скриппс Рисерч Инститьют Гематопоэтические стволовые клетки и способы лечения неоваскулярных заболеваний глаз с их помощью
US20110034546A1 (en) * 2008-04-15 2011-02-10 Genzyme Corporation Methods to produce rod-derived cone viability factor (rdcvf)

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CHOO K.H. et al. Flanking signal and mature peptide residues influence signal peptide cleavage. BMC Bioinformatics. 2008 Dec 12; 9 Suppl 12: S15. *

Also Published As

Publication number Publication date
EP2797613A2 (en) 2014-11-05
BR112014010091B1 (pt) 2022-11-08
HK1202049A1 (en) 2015-09-18
CA2853379A1 (en) 2013-05-02
US9265813B2 (en) 2016-02-23
EP2797613A4 (en) 2015-09-09
JP2015501156A (ja) 2015-01-15
US20160108099A1 (en) 2016-04-21
CN104321069A (zh) 2015-01-28
PL2797613T3 (pl) 2020-06-15
DK2797613T3 (da) 2020-03-02
US20140328821A1 (en) 2014-11-06
EP2797613B1 (en) 2019-12-04
KR20140092320A (ko) 2014-07-23
CA2853379C (en) 2020-11-24
JP6293664B2 (ja) 2018-03-14
AU2012321102C1 (en) 2016-11-10
BR112014010091A2 (pt) 2020-10-27
MX2014004949A (es) 2014-09-12
RU2014121256A (ru) 2015-12-10
WO2013063383A3 (en) 2013-06-20
AU2012321102B2 (en) 2016-07-14
ES2774779T3 (es) 2020-07-22
MX354516B (es) 2018-03-08
AU2012321102A1 (en) 2013-05-16
CN110777124A (zh) 2020-02-11
KR102000141B1 (ko) 2019-07-15
WO2013063383A2 (en) 2013-05-02
US10040835B2 (en) 2018-08-07

Similar Documents

Publication Publication Date Title
US10040835B2 (en) Vectors encoding rod-derived cone viability factor
US10836803B2 (en) Treatment of retinitis pigmentosa
JP2021500071A (ja) ヒト翻訳後修飾ｖｅｇｆ−ｔｒａｐによる眼疾患および転移性大腸がんの処置
CN105980569A (zh) 重组aav-crumbs同源物组合物及治疗lca-8和进行性rp的方法
RS62359B1 (sr) Lečenje amd upotrebom aav sflt-1
US20210347834A1 (en) Gene therapy vector for treating retinitis pigmentosa disease
JP7495756B2 (ja) 医薬の製造におけるＣＹＰ４Ｖ２およびＲｄＣＶＦの使用
AU2022201553B2 (en) Gene therapy to improve vision
WO2022027933A1 (zh) 一种融合蛋白及其用途
WO2025103416A1 (zh) 用于老年相关性黄斑变性的治疗的基因治疗药物
US20230135501A1 (en) Gene therapy
US20240342313A1 (en) Vector encoding rod-derived cone viability factor and human igk signal sequence
HK1202049B (en) Vectors encoding rod-derived cone viability factor
US20260091133A1 (en) Rod-derived cone viability factor fusion protein
Tschernutter Gene therapy for retinal degeneration due to a defect in the retinal pigment epithelium
HK1248595B (en) Gene therapy to improve vision