JP5766389B2 - 脊髄疾患の遺伝子療法 - Google Patents

脊髄疾患の遺伝子療法 Download PDF

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Publication number
JP5766389B2
JP5766389B2 JP2008510154A JP2008510154A JP5766389B2 JP 5766389 B2 JP5766389 B2 JP 5766389B2 JP 2008510154 A JP2008510154 A JP 2008510154A JP 2008510154 A JP2008510154 A JP 2008510154A JP 5766389 B2 JP5766389 B2 JP 5766389B2
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aav
spinal cord
region
mice
transgene
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Japanese (ja)
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JP2008540435A5 (https=
JP2008540435A (ja
Inventor
ジェイムズ・ダッジ
ラムヤ・シハブッディン
マルコ・パッシーニ
セン・チェン
キャサリン・アール・オリオルダン
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Genzyme Corp
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Genzyme Corp
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    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N2750/14041—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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2008510154A 2005-05-02 2006-05-02 脊髄疾患の遺伝子療法 Active JP5766389B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US67721305P 2005-05-02 2005-05-02
US60/677,213 2005-05-02
US79021706P 2006-04-08 2006-04-08
US60/790,217 2006-04-08
PCT/US2006/016943 WO2006119341A2 (en) 2005-05-02 2006-05-02 Gene therapy for spinal cord disorders

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2012282826A Division JP2013082731A (ja) 2005-05-02 2012-12-26 脊髄疾患の遺伝子療法

Publications (3)

Publication Number Publication Date
JP2008540435A JP2008540435A (ja) 2008-11-20
JP2008540435A5 JP2008540435A5 (https=) 2009-06-25
JP5766389B2 true JP5766389B2 (ja) 2015-08-19

Family

ID=37308661

Family Applications (5)

Application Number Title Priority Date Filing Date
JP2008510154A Active JP5766389B2 (ja) 2005-05-02 2006-05-02 脊髄疾患の遺伝子療法
JP2012282826A Pending JP2013082731A (ja) 2005-05-02 2012-12-26 脊髄疾患の遺伝子療法
JP2015063153A Pending JP2015163613A (ja) 2005-05-02 2015-03-25 脊髄疾患の遺伝子療法
JP2017032174A Pending JP2017132774A (ja) 2005-05-02 2017-02-23 脊髄疾患の遺伝子療法
JP2019203155A Active JP6887008B2 (ja) 2005-05-02 2019-11-08 脊髄疾患の遺伝子療法

Family Applications After (4)

Application Number Title Priority Date Filing Date
JP2012282826A Pending JP2013082731A (ja) 2005-05-02 2012-12-26 脊髄疾患の遺伝子療法
JP2015063153A Pending JP2015163613A (ja) 2005-05-02 2015-03-25 脊髄疾患の遺伝子療法
JP2017032174A Pending JP2017132774A (ja) 2005-05-02 2017-02-23 脊髄疾患の遺伝子療法
JP2019203155A Active JP6887008B2 (ja) 2005-05-02 2019-11-08 脊髄疾患の遺伝子療法

Country Status (14)

Country Link
US (2) US10744210B2 (https=)
EP (1) EP1879623B1 (https=)
JP (5) JP5766389B2 (https=)
CN (1) CN105770909A (https=)
AU (1) AU2006242129A1 (https=)
BR (1) BRPI0611540A2 (https=)
CA (1) CA2606490C (https=)
DK (1) DK1879623T3 (https=)
ES (1) ES2394482T3 (https=)
IL (2) IL187023A (https=)
MX (1) MX2007013734A (https=)
PL (1) PL1879623T3 (https=)
PT (1) PT1879623E (https=)
WO (1) WO2006119341A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20160003792A (ko) * 2013-05-01 2016-01-11 젠자임 코포레이션 척수성 근위축증을 치료하기 위한 조성물 및 방법

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US9238150B2 (en) * 2005-07-22 2016-01-19 The Board Of Trustees Of The Leland Stanford Junior University Optical tissue interface method and apparatus for stimulating cells
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US9274099B2 (en) 2005-07-22 2016-03-01 The Board Of Trustees Of The Leland Stanford Junior University Screening test drugs to identify their effects on cell membrane voltage-gated ion channel
US8926959B2 (en) * 2005-07-22 2015-01-06 The Board Of Trustees Of The Leland Stanford Junior University System for optical stimulation of target cells
WO2007146046A2 (en) 2006-06-07 2007-12-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
WO2008086470A1 (en) 2007-01-10 2008-07-17 The Board Of Trustees Of The Leland Stanford Junior University System for optical stimulation of target cells
WO2008101128A1 (en) 2007-02-14 2008-08-21 The Board Of Trustees Of The Leland Stanford Junior University System, method and applications involving identification of biological circuits such as neurological characteristics
WO2008106694A2 (en) 2007-03-01 2008-09-04 The Board Of Trustees Of The Leland Stanford Junior University Systems, methods and compositions for optical stimulation of target cells
EP2019143A1 (en) * 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
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US10434327B2 (en) 2007-10-31 2019-10-08 The Board Of Trustees Of The Leland Stanford Junior University Implantable optical stimulators
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WO2009148946A2 (en) * 2008-05-29 2009-12-10 The Board Of Trustees Of The Leland Stanford Junior University Cell line, system and method for optical control of secondary messengers
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JP2011525130A (ja) 2008-06-17 2011-09-15 ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティ 光伝送素子を用いた標的細胞の光刺激のための方法、システム、および装置
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JP5879256B2 (ja) * 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
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CN106376525A (zh) 2010-11-05 2017-02-08 斯坦福大学托管董事会 记忆功能的控制和表征
CN106422081B (zh) 2010-11-05 2019-06-21 斯坦福大学托管董事会 用于光遗传学方法的光的上转换
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Publication number Priority date Publication date Assignee Title
KR20160003792A (ko) * 2013-05-01 2016-01-11 젠자임 코포레이션 척수성 근위축증을 치료하기 위한 조성물 및 방법
KR102116378B1 (ko) * 2013-05-01 2020-06-01 젠자임 코포레이션 척수성 근위축증을 치료하기 위한 조성물 및 방법

Also Published As

Publication number Publication date
JP2015163613A (ja) 2015-09-10
WO2006119341A2 (en) 2006-11-09
EP1879623A2 (en) 2008-01-23
IL187023A (en) 2015-10-29
MX2007013734A (es) 2008-03-14
JP2013082731A (ja) 2013-05-09
CN105770909A (zh) 2016-07-20
BRPI0611540A2 (pt) 2010-09-21
US20210008227A1 (en) 2021-01-14
EP1879623B1 (en) 2012-10-03
JP6887008B2 (ja) 2021-06-16
CA2606490C (en) 2018-02-06
US20090069261A1 (en) 2009-03-12
IL187023A0 (en) 2008-02-09
EP1879623A4 (en) 2009-03-25
CA2606490A1 (en) 2006-11-09
IL237658A0 (en) 2015-04-30
JP2017132774A (ja) 2017-08-03
DK1879623T3 (da) 2012-12-17
AU2006242129A1 (en) 2006-11-09
WO2006119341A3 (en) 2007-06-28
PL1879623T3 (pl) 2013-03-29
JP2020050660A (ja) 2020-04-02
US10744210B2 (en) 2020-08-18
PT1879623E (pt) 2012-12-20
JP2008540435A (ja) 2008-11-20
ES2394482T3 (es) 2013-02-01

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