CN102821781A - 治疗帕金森氏病和脑多巴胺能神经元的其它病症的方法 - Google Patents

治疗帕金森氏病和脑多巴胺能神经元的其它病症的方法 Download PDF

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CN102821781A
CN102821781A CN2011800174012A CN201180017401A CN102821781A CN 102821781 A CN102821781 A CN 102821781A CN 2011800174012 A CN2011800174012 A CN 2011800174012A CN 201180017401 A CN201180017401 A CN 201180017401A CN 102821781 A CN102821781 A CN 102821781A
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growth factor
nerve growth
disease
substantia nigra
brain
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Chinese (zh)
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R·T·巴尔图斯
J·斯费尔特
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Ceregene Inc
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Ceregene Inc
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    • C07K14/475—Growth factors; Growth regulators
    • C07K14/48—Nerve growth factor [NGF]
    • A—HUMAN NECESSITIES
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    • 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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
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    • 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
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    • 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
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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    • 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)
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  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN2011800174012A 2010-04-02 2011-04-01 治疗帕金森氏病和脑多巴胺能神经元的其它病症的方法 Pending CN102821781A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US32065410P 2010-04-02 2010-04-02
US61/320,654 2010-04-02
PCT/US2011/031027 WO2011123842A2 (en) 2010-04-02 2011-04-01 Methods for treating parkinson's disease and other disorders of dopaminergic neurons of the brain

Publications (1)

Publication Number Publication Date
CN102821781A true CN102821781A (zh) 2012-12-12

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US (1) US20120082650A1 (enExample)
EP (1) EP2552473A4 (enExample)
JP (1) JP2013523776A (enExample)
CN (1) CN102821781A (enExample)
AU (1) AU2011235890B2 (enExample)
CA (1) CA2793692A1 (enExample)
WO (1) WO2011123842A2 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108024544B (zh) * 2015-07-13 2022-04-29 桑格摩生物治疗股份有限公司 用于核酸酶介导的基因组工程化的递送方法及组合物
AU2019261646A1 (en) 2018-04-27 2020-11-12 Spacecraft Seven, Llc Gene therapy for CNS degeneration
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
KR20250142386A (ko) 2023-02-03 2025-09-30 얀센 파마슈티카 엔브이 파킨슨병에 사용하기 위한 유전자 요법 벡터

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6800281B2 (en) * 2000-11-09 2004-10-05 Oxford Biomedica (Uk) Limited Lentiviral-mediated growth factor gene therapy for neurodegenerative diseases
CN1897977A (zh) * 2003-10-20 2007-01-17 Ns基因公司 帕金森氏病的体内基因治疗

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
BARTUS RT ET AL.: "Bioactivity of AAV2-neurturin gene therapy (CERE-120):difference between parkinson"s disease and nonhuman primate brains", 《MOV DISORD》 *
HADACZEK P ET AL.: "The "perivascular pump" driven by arterial pulsation is a powerful mechanism for the distribution of therapeutic molecules within the brain", 《MOL THER》 *
HERZOG CD ET AL.: "Transgene expression,bioactivity,and safety of CERE-120(AAV2-neurturin) following delivery to the monkey striatum", 《MOL THER》 *
JOHNSTON LC ET AL.,: "Clinically relevant effects of convection-enhanced delivery of AAV2-DGNF on the dopaminergic nigrostriatal pathway in aged thesus monkeys", 《HUMAN GENE THERAPY》 *
SLEVIN JT ET AL.: "Unilateral intraputaminal glial cell-line derived neurotrophic factor in patients with parkinson disease:response to 1 year each of treatment and withdrawal", 《NEUROSURG FOCUS》 *

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Publication number Publication date
CA2793692A1 (en) 2011-10-06
JP2013523776A (ja) 2013-06-17
EP2552473A2 (en) 2013-02-06
WO2011123842A9 (en) 2012-01-12
AU2011235890B2 (en) 2014-07-31
WO2011123842A2 (en) 2011-10-06
AU2011235890A1 (en) 2012-09-27
EP2552473A4 (en) 2013-10-23
US20120082650A1 (en) 2012-04-05

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Application publication date: 20121212