JP2013523776A - パーキンソン病および脳のドーパミン作動性ニューロンのその他の障害を処置する方法 - Google Patents

パーキンソン病および脳のドーパミン作動性ニューロンのその他の障害を処置する方法 Download PDF

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JP2013523776A
JP2013523776A JP2013502910A JP2013502910A JP2013523776A JP 2013523776 A JP2013523776 A JP 2013523776A JP 2013502910 A JP2013502910 A JP 2013502910A JP 2013502910 A JP2013502910 A JP 2013502910A JP 2013523776 A JP2013523776 A JP 2013523776A
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growth factor
nerve growth
disease
substantia nigra
striatum
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JP2013523776A5 (enExample
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レイモンド ティー. バータス
ジョアオ シフェール
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セレジーン インコーポレイテッド
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • C07K14/48Nerve growth factor [NGF]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/185Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025Uses 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)
  • Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Biotechnology (AREA)
  • Zoology (AREA)
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  • Molecular Biology (AREA)
  • Psychology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2013502910A 2010-04-02 2011-04-01 パーキンソン病および脳のドーパミン作動性ニューロンのその他の障害を処置する方法 Withdrawn JP2013523776A (ja)

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

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JP2013523776A true JP2013523776A (ja) 2013-06-17
JP2013523776A5 JP2013523776A5 (enExample) 2014-05-15

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JP2013502910A Withdrawn JP2013523776A (ja) 2010-04-02 2011-04-01 パーキンソン病および脳のドーパミン作動性ニューロンのその他の障害を処置する方法

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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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021045166A (ja) * 2015-07-13 2021-03-25 サンガモ セラピューティクス, インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020021903A2 (pt) 2018-04-27 2021-03-02 Rocket Pharmaceuticals, Ltd. terapia genética para degeneração do snc
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
IL322484A (en) 2023-02-03 2025-09-01 Janssen Pharmaceutica Nv Gene therapy vectors for use in Parkinson's disease

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
US20060239966A1 (en) * 2003-10-20 2006-10-26 Tornoee Jens In vivo gene therapy of parkinson's disease

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021045166A (ja) * 2015-07-13 2021-03-25 サンガモ セラピューティクス, インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物
JP7348160B2 (ja) 2015-07-13 2023-09-20 サンガモ セラピューティクス, インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

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CN102821781A (zh) 2012-12-12
CA2793692A1 (en) 2011-10-06
WO2011123842A2 (en) 2011-10-06
EP2552473A2 (en) 2013-02-06
WO2011123842A9 (en) 2012-01-12
AU2011235890B2 (en) 2014-07-31
AU2011235890A1 (en) 2012-09-27
US20120082650A1 (en) 2012-04-05
EP2552473A4 (en) 2013-10-23

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