CN101980668A - 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器 - Google Patents

通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器 Download PDF

Info

Publication number
CN101980668A
CN101980668A CN2009801043603A CN200980104360A CN101980668A CN 101980668 A CN101980668 A CN 101980668A CN 2009801043603 A CN2009801043603 A CN 2009801043603A CN 200980104360 A CN200980104360 A CN 200980104360A CN 101980668 A CN101980668 A CN 101980668A
Authority
CN
China
Prior art keywords
cell
aav
retina
eye
vector
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN2009801043603A
Other languages
English (en)
Chinese (zh)
Inventor
R·T·巴尔图斯
K·M·毕晓普
M·加斯米
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ceregene Inc
Original Assignee
Ceregene Inc
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 Ceregene Inc filed Critical Ceregene Inc
Publication of CN101980668A publication Critical patent/CN101980668A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • A61K9/0051—Ocular inserts or implants
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN2009801043603A 2008-02-07 2009-02-05 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器 Pending CN101980668A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US2699008P 2008-02-07 2008-02-07
US61/026,990 2008-02-07
US9322808P 2008-08-29 2008-08-29
US61/093,228 2008-08-29
PCT/US2009/033275 WO2009100253A1 (en) 2008-02-07 2009-02-05 Rescue of photoreceptors by intravitreal administation of an expression vector encoding a therapeutic protein

Publications (1)

Publication Number Publication Date
CN101980668A true CN101980668A (zh) 2011-02-23

Family

ID=40939054

Family Applications (1)

Application Number Title Priority Date Filing Date
CN2009801043603A Pending CN101980668A (zh) 2008-02-07 2009-02-05 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器

Country Status (7)

Country Link
US (2) US8242093B2 (https=)
EP (1) EP2247258A4 (https=)
JP (1) JP2011511090A (https=)
CN (1) CN101980668A (https=)
AU (1) AU2009212309B2 (https=)
CA (1) CA2713755A1 (https=)
WO (1) WO2009100253A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107937341A (zh) * 2011-02-25 2018-04-20 国立研究开发法人理化学研究所 视网膜色素上皮细胞片层的生产方法

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2009212309B2 (en) * 2008-02-07 2014-04-03 Ceregene, Inc. Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method
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
WO2015044704A1 (en) * 2013-09-30 2015-04-02 Sanofi Use of neuroglobin agonist for preventing or treating mitochondrial rcci and/or rcciii deficiency disease
AU2014359136B2 (en) * 2013-12-06 2020-06-25 Centre Hospitalier Universitaire De Montpellier Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
WO2016164920A1 (en) * 2015-04-09 2016-10-13 Cornell University Gene therapy to prevent reactions to allergens
AU2016363830C1 (en) * 2015-12-04 2022-03-24 Centre National De La Recherche Scientifique Promoters and uses thereof
AU2017256910B2 (en) * 2016-04-29 2022-04-07 Centre National De La Recherche Scientifique Optogenetic visual restoration using Chrimson
PL239642B1 (pl) * 2018-08-16 2021-12-20 Pomorski Univ Medyczny W Szczecinie Kompozycja do doszklistkowego podawania białka leczniczego
CA3199230A1 (en) 2020-10-22 2022-04-28 Visgenx, Inc. Elovl2 constructs for human gene therapy
AU2023390868A1 (en) * 2022-12-05 2025-06-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Pigment epithelium-derived factor peptides and use for treating retinal degeneration

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020194630A1 (en) * 1999-03-15 2002-12-19 Manning William C. Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US20050208103A1 (en) * 1999-01-05 2005-09-22 Adamis Anthony P Targeted transscleral controlled release drug delivery to the retina and choroid
US20060188481A1 (en) * 2005-02-22 2006-08-24 Saitama Medical School Methods for prophylactically or therapeutically treating an animal for an ocular-related disorder

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5932479A (en) 1988-09-26 1999-08-03 Auburn University Genetic engineering of plant chloroplasts
DE69817180D1 (de) * 1997-06-11 2003-09-18 Acorda Therapeutics Hawthorne Zns neuroregenerative zusammensetzungen und deren verwendungen
AU3755900A (en) * 1999-03-15 2000-10-04 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
AU2001241809A1 (en) 2000-02-29 2001-09-12 Auburn University Genetic engineering of drought tolerance via a plastid genome
CA2402066C (en) 2000-03-01 2015-09-29 Auburn University Plastid transformation vectors for expressing human proteins in plants
US7354760B2 (en) 2001-12-26 2008-04-08 University Of Central Florida Research Foundation, Inc. Expression of protective antigens in transgenic chloroplasts
AU2003253781A1 (en) 2002-07-03 2004-01-23 University Of Central Florida Expression of human interferon in transgenic chloroplasts
US20100172871A1 (en) 2005-02-17 2010-07-08 Flannery John G Muller Cell Specific Gene Therapy
CA2598029A1 (en) 2005-02-24 2006-10-05 The Scripps Research Institute Isolated myeloid-like bone marrow cell populations and methods of treatment therewith
US20080274143A1 (en) 2005-05-27 2008-11-06 Henry Daniell Chloroplasts Engineering to Express Pharmaceutical Proteins
AU2009212309B2 (en) * 2008-02-07 2014-04-03 Ceregene, Inc. Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050208103A1 (en) * 1999-01-05 2005-09-22 Adamis Anthony P Targeted transscleral controlled release drug delivery to the retina and choroid
US20020194630A1 (en) * 1999-03-15 2002-12-19 Manning William C. Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US20060188481A1 (en) * 2005-02-22 2006-08-24 Saitama Medical School Methods for prophylactically or therapeutically treating an animal for an ocular-related disorder

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107937341A (zh) * 2011-02-25 2018-04-20 国立研究开发法人理化学研究所 视网膜色素上皮细胞片层的生产方法

Also Published As

Publication number Publication date
AU2009212309A1 (en) 2009-08-13
AU2009212309B2 (en) 2014-04-03
JP2011511090A (ja) 2011-04-07
EP2247258A1 (en) 2010-11-10
WO2009100253A8 (en) 2010-08-19
US20090202505A1 (en) 2009-08-13
US20120316112A1 (en) 2012-12-13
US8420619B2 (en) 2013-04-16
US8242093B2 (en) 2012-08-14
WO2009100253A1 (en) 2009-08-13
CA2713755A1 (en) 2009-08-13
EP2247258A4 (en) 2012-01-18

Similar Documents

Publication Publication Date Title
CN101980668A (zh) 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器
Lau et al. Retinal degeneration is slowed in transgenic rats by AAV-mediated delivery of FGF-2
Harvey et al. Intravitreal injection of adeno-associated viral vectors results in the transduction of different types of retinal neurons in neonatal and adult rats: a comparison with lentiviral vectors
JP7097398B2 (ja) ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター
US20210388386A1 (en) Synp57 (proa14), a promoter for the specific expression of genes in photoreceptors
Thanos et al. Delivery of neurotrophic factors and therapeutic proteins for retinal diseases
TW201350581A (zh) 治療視網膜營養性萎縮之病毒載體
CN105770909A (zh) 脊髓失调症的基因治疗
Igarashi et al. Tyrosine triple mutated AAV2-BDNF gene therapy in a rat model of transient IOP elevation
Thumann Prospectives for gene therapy of retinal degenerations
US20230233709A1 (en) Gene therapy for ocular disorders
Pang et al. Efficiency of lentiviral transduction during development in normal and rd mice
US11845952B2 (en) Adeno-associated virus vector
JP2024105661A (ja) 眼疾患に対するａｄｅｎｏ随伴ウイルス連ウイルスベクター媒介遺伝子治療
US20250171738A1 (en) Process for producing cone photoreceptor cells
Cronin et al. Retinal degenerations: from cell signaling to cell therapy; pre-clinical and clinical issues
CN111705069A (zh) 一种多神经营养因子联合表达载体及其应用
Dureau et al. Quantitative analysis of subretinal injections in the rat
Chacón-Camacho et al. Gene therapy for hereditary ophthalmological diseases: Advances and future perspectives
KR20250083388A (ko) 신규한 케모카인-유사 단백질 단편
CN118146317A (zh) 一种腺相关病毒衣壳蛋白及其用途
CN118272441A (zh) 组合元件，表达载体及其应用
CN111850043A (zh) Ecel1重组腺相关病毒载体及应用

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C02 Deemed withdrawal of patent application after publication (patent law 2001)
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20110223