JP2021533126A - 臓器機能を制御するための遺伝子治療方法 - Google Patents

臓器機能を制御するための遺伝子治療方法 Download PDF

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JP2021533126A
JP2021533126A JP2021505674A JP2021505674A JP2021533126A JP 2021533126 A JP2021533126 A JP 2021533126A JP 2021505674 A JP2021505674 A JP 2021505674A JP 2021505674 A JP2021505674 A JP 2021505674A JP 2021533126 A JP2021533126 A JP 2021533126A
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vector
aav
viral vector
nerve
gene
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JP2021533126A5 (https=
JPWO2020028466A5 (https=
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マイケル ジー． カプリット
ロベルタ マロンジュ
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Cornell University
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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
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1787—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/14—Antitussive 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
    • 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
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • 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
    • C12N2750/14111—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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Pulmonology (AREA)
  • Neurosurgery (AREA)
  • Otolaryngology (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2021505674A 2018-07-31 2019-07-31 臓器機能を制御するための遺伝子治療方法 Pending JP2021533126A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024001612A JP2024041871A (ja) 2018-07-31 2024-01-10 臓器機能を制御するための遺伝子治療方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862712669P 2018-07-31 2018-07-31
US62/712,669 2018-07-31
PCT/US2019/044290 WO2020028466A1 (en) 2018-07-31 2019-07-31 Gene therapy methods to control organ function

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JP2021533126A true JP2021533126A (ja) 2021-12-02
JP2021533126A5 JP2021533126A5 (https=) 2022-08-01
JPWO2020028466A5 JPWO2020028466A5 (https=) 2022-08-01

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JP2024001612A Pending JP2024041871A (ja) 2018-07-31 2024-01-10 臓器機能を制御するための遺伝子治療方法

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US (1) US20210301306A1 (https=)
EP (1) EP3829718A4 (https=)
JP (2) JP2021533126A (https=)
KR (1) KR20210052450A (https=)
CN (1) CN113301956B (https=)
AU (1) AU2019315445A1 (https=)
BR (1) BR112021001878A2 (https=)
CA (1) CA3108324A1 (https=)
EA (1) EA202190358A1 (https=)
IL (1) IL280531A (https=)
MX (1) MX2021001336A (https=)
PH (1) PH12021550243A1 (https=)
SG (1) SG11202101042SA (https=)
WO (1) WO2020028466A1 (https=)

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WO2013134543A1 (en) 2012-03-08 2013-09-12 Medtronic Ardian Luxembourg Sarl Immune system neuromodulation and associated systems and methods
JP2017536187A (ja) 2014-12-03 2017-12-07 メタベンション インコーポレイテッド 神経または他の組織を調節するためのシステムおよび方法
US10524859B2 (en) 2016-06-07 2020-01-07 Metavention, Inc. Therapeutic tissue modulation devices and methods
CN115707773A (zh) * 2021-08-20 2023-02-21 中国科学院深圳先进技术研究院 一种利用交感神经控制血管内皮细胞异质性的方法
WO2023019565A1 (zh) * 2021-08-20 2023-02-23 中国科学院深圳先进技术研究院 一种利用交感神经控制血管内皮细胞异质性的方法
CN116019909B (zh) * 2022-09-16 2025-07-29 上海市第十人民医院 一种CRISPR/Cas9纳米系统
CN116115758B (zh) * 2022-11-22 2024-12-03 上海市内分泌代谢病研究所 Gabaa受体作为靶点在制备或筛选降血脂、治疗肥胖和/或改善代谢的药物中的应用
CN120699131B (zh) * 2024-12-04 2025-12-09 良渚实验室 治疗帕金森病的化学遗传学基因治疗新方案

Citations (4)

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WO2017049252A1 (en) * 2015-09-17 2017-03-23 Switch Bio, Inc. Compositions and methods for treating neurological disorders
WO2017153995A1 (en) * 2016-03-09 2017-09-14 Assaf Fadi Using dreadd for neuronal modulation in treating neuronal diseases
JP2017534601A (ja) * 2014-10-03 2017-11-24 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2018008770A1 (ja) * 2016-07-06 2018-01-11 国立研究開発法人国立循環器病研究センター 末梢神経を操作する方法、脳および脊髄を除く臓器などにおいて神経細胞機能を変化させる機能を発生させる方法、および新規疾病の予防または治療方法、ならびに末梢神経投与用医薬

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WO2018008770A1 (ja) * 2016-07-06 2018-01-11 国立研究開発法人国立循環器病研究センター 末梢神経を操作する方法、脳および脊髄を除く臓器などにおいて神経細胞機能を変化させる機能を発生させる方法、および新規疾病の予防または治療方法、ならびに末梢神経投与用医薬

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Publication number Publication date
EP3829718A1 (en) 2021-06-09
CN113301956B (zh) 2025-06-24
CA3108324A1 (en) 2020-02-06
EA202190358A1 (ru) 2021-05-04
PH12021550243A1 (en) 2021-11-22
KR20210052450A (ko) 2021-05-10
WO2020028466A1 (en) 2020-02-06
CN113301956A (zh) 2021-08-24
US20210301306A1 (en) 2021-09-30
BR112021001878A2 (pt) 2021-04-27
EP3829718A4 (en) 2022-06-22
SG11202101042SA (en) 2021-02-25
AU2019315445A1 (en) 2021-03-18
MX2021001336A (es) 2021-09-10
JP2024041871A (ja) 2024-03-27
IL280531A (en) 2021-03-25

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