JP2011511090A - 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 - Google Patents

治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 Download PDF

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JP2011511090A
JP2011511090A JP2010546007A JP2010546007A JP2011511090A JP 2011511090 A JP2011511090 A JP 2011511090A JP 2010546007 A JP2010546007 A JP 2010546007A JP 2010546007 A JP2010546007 A JP 2010546007A JP 2011511090 A JP2011511090 A JP 2011511090A
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cells
aav
vector
retinal
eye
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JP2011511090A5 (enExample
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レイモンド ティー． バータス
キャシー エム． ビショップ
メディ ガスミ
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セレジーン インコーポレイテッド
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    • 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

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  • 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)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2010546007A 2008-02-07 2009-02-05 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 Ceased JP2011511090A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US2699008P 2008-02-07 2008-02-07
US9322808P 2008-08-29 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 (2)

Publication Number Publication Date
JP2011511090A true JP2011511090A (ja) 2011-04-07
JP2011511090A5 JP2011511090A5 (enExample) 2013-03-28

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JP2010546007A Ceased JP2011511090A (ja) 2008-02-07 2009-02-05 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法

Country Status (7)

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US (2) US8242093B2 (enExample)
EP (1) EP2247258A4 (enExample)
JP (1) JP2011511090A (enExample)
CN (1) CN101980668A (enExample)
AU (1) AU2009212309B2 (enExample)
CA (1) CA2713755A1 (enExample)
WO (1) WO2009100253A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2713755A1 (en) * 2008-02-07 2009-08-13 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
BR112013021514A2 (pt) * 2011-02-25 2020-09-29 Riken método de produção de camada de célula epitelial de pigmento retinal
CN105120901A (zh) * 2012-07-11 2015-12-02 宾夕法尼亚大学托管会 Rpgr x染色体连锁视网膜退化的aav介导的基因治疗
US20160256571A1 (en) * 2013-09-30 2016-09-08 Sanofi Invention
KR102281881B1 (ko) * 2013-12-06 2021-07-27 엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔 (인쎄름) 대상체의 망막 색소 상피에서 목적 폴리뉴클레오티드를 발현시키기 위한 방법 및 약학적 조성물
JP6878301B2 (ja) * 2015-04-09 2021-05-26 コーネル ユニヴァーシティー アレルゲンに対する反応を予防するための遺伝子治療
JP6966442B2 (ja) * 2015-12-04 2021-11-17 ソルボンヌ・ユニヴェルシテSorbonne Universite プロモーター及びその使用
EP3448411A1 (en) * 2016-04-29 2019-03-06 Gensight Biologics SA Optogenetic visual restoration using chrimson
PL239642B1 (pl) * 2018-08-16 2021-12-20 Pomorski Univ Medyczny W Szczecinie Kompozycja do doszklistkowego podawania białka leczniczego
JP2023546751A (ja) 2020-10-22 2023-11-07 ヴィスゲニックス・インコーポレイテッド ヒト遺伝子療法のためのｅｌｏｖｌ２コンストラクト
CN117062629A (zh) * 2021-02-26 2023-11-14 西奈山伊坎医学院 用于减少视网膜神经节细胞的变性的方法
JP2025540499A (ja) * 2022-12-05 2025-12-11 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 色素上皮由来因子ペプチドおよび網膜変性症を処置するための使用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002512627A (ja) * 1997-06-11 2002-04-23 アコーダ セラピーティックス Ｃｎｓ神経再生組成物及びその使用方法
JP2002539176A (ja) * 1999-03-15 2002-11-19 カイロン コーポレイション 眼の疾患を処置または予防するための組換え遺伝子送達ベクターの使用

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* Cited by examiner, † Cited by third party
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US5932479A (en) 1988-09-26 1999-08-03 Auburn University Genetic engineering of plant chloroplasts
JP2002534139A (ja) * 1999-01-05 2002-10-15 マサチューセッツ・アイ・アンド・イア・インファーマリー 網膜と脈絡膜への経強膜徐放性薬剤標的送達
US6943153B1 (en) * 1999-03-15 2005-09-13 The Regents Of The University Of California 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
EP1274846A4 (en) 2000-03-01 2004-09-29 Univ Auburn PLASTIDE TRANSFORMATION VECTOR FOR THE EXPRESSION OF 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
WO2006101634A1 (en) 2005-02-17 2006-09-28 The Regents Of The University Of California Müller cell specific gene therapy
US20060188481A1 (en) * 2005-02-22 2006-08-24 Saitama Medical School Methods for prophylactically or therapeutically treating an animal for an ocular-related disorder
RU2418856C2 (ru) 2005-02-24 2011-05-20 Дзе Скриппс Рисерч Инститьют Изолированные популяции миелоидоподобных клеток костного мозга и способы лечения с ними
US20080274143A1 (en) 2005-05-27 2008-11-06 Henry Daniell Chloroplasts Engineering to Express Pharmaceutical Proteins
CA2713755A1 (en) * 2008-02-07 2009-08-13 Ceregene, Inc. Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002512627A (ja) * 1997-06-11 2002-04-23 アコーダ セラピーティックス Ｃｎｓ神経再生組成物及びその使用方法
JP2002539176A (ja) * 1999-03-15 2002-11-19 カイロン コーポレイション 眼の疾患を処置または予防するための組換え遺伝子送達ベクターの使用

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Publication number Publication date
EP2247258A4 (en) 2012-01-18
US8420619B2 (en) 2013-04-16
CN101980668A (zh) 2011-02-23
WO2009100253A1 (en) 2009-08-13
AU2009212309A1 (en) 2009-08-13
US20090202505A1 (en) 2009-08-13
CA2713755A1 (en) 2009-08-13
US20120316112A1 (en) 2012-12-13
US8242093B2 (en) 2012-08-14
EP2247258A1 (en) 2010-11-10
AU2009212309B2 (en) 2014-04-03
WO2009100253A8 (en) 2010-08-19

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