JP2003522160A - 眼関連疾患の治療のための遺伝子治療 - Google Patents

眼関連疾患の治療のための遺伝子治療

Info

Publication number
JP2003522160A
JP2003522160A JP2001557602A JP2001557602A JP2003522160A JP 2003522160 A JP2003522160 A JP 2003522160A JP 2001557602 A JP2001557602 A JP 2001557602A JP 2001557602 A JP2001557602 A JP 2001557602A JP 2003522160 A JP2003522160 A JP 2003522160A
Authority
JP
Japan
Prior art keywords
nucleic acid
acid sequence
vector
expression vector
sequence encoding
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
JP2001557602A
Other languages
English (en)
Japanese (ja)
Other versions
JP2003522160A5 (enExample
Inventor
コウベスディ、イムリ
イー． ブロー、ダグラス
エル． マクヴェイ、ダンカン
ウェイ、リサ
Original Assignee
ジェンベク、インコーポレイティッド
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
Priority claimed from US09/599,997 external-priority patent/US6821775B1/en
Application filed by ジェンベク、インコーポレイティッド filed Critical ジェンベク、インコーポレイティッド
Publication of JP2003522160A publication Critical patent/JP2003522160A/ja
Publication of JP2003522160A5 publication Critical patent/JP2003522160A5/ja
Pending legal-status Critical Current

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
    • A61K48/0075—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
    • 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
    • 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
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2001557602A 2000-02-11 2001-02-09 眼関連疾患の治療のための遺伝子治療 Pending JP2003522160A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US18174300P 2000-02-11 2000-02-11
US60/181,743 2000-02-11
US09/599,997 US6821775B1 (en) 2000-02-11 2000-06-23 Viral vector encoding pigment epithelium-derived factor
US09/599,997 2000-06-23
US22833700P 2000-08-28 2000-08-28
US60/228,337 2000-08-28
PCT/US2001/004203 WO2001058494A2 (en) 2000-02-11 2001-02-09 Gene therapy for treating ocular-related disorders

Publications (2)

Publication Number Publication Date
JP2003522160A true JP2003522160A (ja) 2003-07-22
JP2003522160A5 JP2003522160A5 (enExample) 2008-04-10

Family

ID=27391455

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2001557602A Pending JP2003522160A (ja) 2000-02-11 2001-02-09 眼関連疾患の治療のための遺伝子治療

Country Status (5)

Country Link
EP (1) EP1253948A2 (enExample)
JP (1) JP2003522160A (enExample)
AU (1) AU780634C (enExample)
CA (1) CA2398571A1 (enExample)
WO (1) WO2001058494A2 (enExample)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007291009A (ja) * 2006-04-25 2007-11-08 Seitai Shigen Laboratory Inc 血管内膜肥厚抑制剤
JP2008538370A (ja) * 2005-04-18 2008-10-23 アンスティテュー・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル・（イ・エヌ・エス・ウ・エール・エム） 対象の眼球に対して治療用生成物を送達するための改良型方法及び装置
JP2015522590A (ja) * 2012-06-25 2015-08-06 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドＴｈｅ Ｂｒｉｇｈａｍ ａｎｄ Ｗｏｍｅｎ’ｓ Ｈｏｓｐｉｔａｌ， Ｉｎｃ． 標的化治療薬
US9790264B2 (en) 2012-06-25 2017-10-17 The Brigham And Women's Hospital, Inc. Compounds and methods for modulating pharmacokinetics
JP2021511834A (ja) * 2018-01-23 2021-05-13 サザン・アイ・エクイップメント 発現ベクター及び方法

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7470659B2 (en) 2001-12-07 2008-12-30 The Regents Of The University Of California Methods to increase reverse cholesterol transport in the retinal pigment epithelium (RPE) and Bruch's membrane (BM)
AU2003297607A1 (en) * 2002-12-02 2004-06-23 Genvec, Inc Materials and methods for treating ocular-related disorders
US20040166091A1 (en) * 2003-02-24 2004-08-26 Genvec, Inc. Materials and methods for treating disorders of the ear
CA2588093A1 (en) 2004-11-16 2006-05-26 Rony Seger Variants of pigment epithelium derived factor and uses thereof
WO2009126894A2 (en) * 2008-04-11 2009-10-15 Massachusetts Eye And Ear Infirmary Methods and compositions for the diagnosis and treatment of angiogenic disorders
US10385112B2 (en) 2013-03-14 2019-08-20 Asklepios Biopharmaceutical, Inc. Modified soluble VEGF receptor-1 genes and vectors for gene therapy
TWI710635B (zh) 2014-10-09 2020-11-21 美商珍維克公司 編碼人類無調同源物-1(hath1)之腺病毒載體
KR102785135B1 (ko) 2018-06-11 2025-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 눈 질환을 치료하는 탈메틸화
CA3104472C (en) * 2018-06-19 2025-12-16 Cella Therapeutics, Llc Drug delivery systems comprising active pharmaceutical ingredients and a sustained delevery component
AU2019288296B2 (en) * 2018-06-19 2022-11-24 Cella Therapeutics, Llc Sustained-release drug delivery systems comprising an intraocular pressure lowering agent, a CNP compound, an NPR-B compound, a Tie-2 agonist, or neurotrophic agent for use for treating glaucoma or ocular hypertension
JP7769388B2 (ja) 2019-12-12 2025-11-13 ティン セラピューティクス，インコーポレイテッド 聴覚損失の予防及び治療のための組成物及び方法
KR20220118499A (ko) * 2019-12-18 2022-08-25 셀라 테라퓨틱스, 엘엘씨 신경영양제, 세포자멸 신호전달 단편 억제제(FAS) 또는 FAS 리간드(FASL) 억제제, 종양 괴사 인자-α(TNF-α) 또는 TNF 수용체 억제제, 미토콘드리아 펩티드, 올리고뉴클레오티드, 케모카인 억제제 또는 시스테인-아스파틱 프로테아제 억제제를 포함하는 약물 전달 시스템

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997034586A2 (en) * 1996-03-22 1997-09-25 Cytotherapeutics, Inc. Device and method for treating ophthalmic diseases
WO1999004806A1 (en) * 1997-07-23 1999-02-04 Northwestern University Methods and compositions for inhibiting angiogenesis
WO1999015686A1 (en) * 1997-09-23 1999-04-01 Genvec, Inc. Plasmids for construction of eukaryotic viral vectors

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5840686A (en) * 1992-09-24 1998-11-24 The United States Of America As Represented By The Department Of Health And Human Services Pigment epithelium-derived factor: characterization of its novel biological activity and sequences encoding and expressing the protein and methods of use
US6319687B1 (en) * 1992-09-24 2001-11-20 The United States Of America As Represented By The Department Of Health And Human Services Pigment epithelium-derived factor: characterization, genomic organization and sequence of PEDF gene
US5461749A (en) * 1994-05-31 1995-10-31 Minnesota Mining And Manufacturing Company Floor mop and cleaning system
WO1999026480A1 (en) * 1997-11-20 1999-06-03 Genetix Pharmaceuticals, Inc. Anti-angiogenic gene therapy vectors and their use in treating angiogenesis-related diseases
AU3755900A (en) * 1999-03-15 2000-10-04 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997034586A2 (en) * 1996-03-22 1997-09-25 Cytotherapeutics, Inc. Device and method for treating ophthalmic diseases
WO1999004806A1 (en) * 1997-07-23 1999-02-04 Northwestern University Methods and compositions for inhibiting angiogenesis
WO1999015686A1 (en) * 1997-09-23 1999-04-01 Genvec, Inc. Plasmids for construction of eukaryotic viral vectors

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008538370A (ja) * 2005-04-18 2008-10-23 アンスティテュー・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル・（イ・エヌ・エス・ウ・エール・エム） 対象の眼球に対して治療用生成物を送達するための改良型方法及び装置
JP2007291009A (ja) * 2006-04-25 2007-11-08 Seitai Shigen Laboratory Inc 血管内膜肥厚抑制剤
JP2015522590A (ja) * 2012-06-25 2015-08-06 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドＴｈｅ Ｂｒｉｇｈａｍ ａｎｄ Ｗｏｍｅｎ’ｓ Ｈｏｓｐｉｔａｌ， Ｉｎｃ． 標的化治療薬
US9421245B2 (en) 2012-06-25 2016-08-23 The Brigham And Women's Hospital, Inc. Targeted therapeutics
US9790264B2 (en) 2012-06-25 2017-10-17 The Brigham And Women's Hospital, Inc. Compounds and methods for modulating pharmacokinetics
JP2021511834A (ja) * 2018-01-23 2021-05-13 サザン・アイ・エクイップメント 発現ベクター及び方法

Also Published As

Publication number Publication date
WO2001058494A3 (en) 2002-04-04
CA2398571A1 (en) 2001-08-16
AU780634B2 (en) 2005-04-07
AU3681801A (en) 2001-08-20
AU780634C (en) 2006-02-23
EP1253948A2 (en) 2002-11-06
WO2001058494A2 (en) 2001-08-16

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