EA202190358A1 - Способы генной терапии для контроля функции органов - Google Patents

Способы генной терапии для контроля функции органов

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Publication number
EA202190358A1
EA202190358A1 EA202190358A EA202190358A EA202190358A1 EA 202190358 A1 EA202190358 A1 EA 202190358A1 EA 202190358 A EA202190358 A EA 202190358A EA 202190358 A EA202190358 A EA 202190358A EA 202190358 A1 EA202190358 A1 EA 202190358A1
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Eurasian Patent Office
Prior art keywords
organ
function
control
organ function
gene therapy
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Application number
EA202190358A
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English (en)
Inventor
Майкл Дж. Каплитт
Роберта Маронгиу
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Корнелл Юниверсити
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Publication of EA202190358A1 publication Critical patent/EA202190358A1/ru

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    • 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
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    • 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
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • 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
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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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  • Health & Medical Sciences (AREA)
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  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Предложены способы и композиции для контроля функции висцерального органа. Например, указанные способы и композиции применимы для предотвращения, ингибирования или лечения заболевания в результате контроля, например регулирования функции органа. В одном варианте осуществления в орган доставляют вирусные векторы, и вирус инфицирует нерв, который регулирует функцию органа. В одном варианте осуществления вирусный вектор представляет собой ретроградный вектор. В одном варианте осуществления вирусный вектор кодирует генный продукт, активность которого контролируют экзогенно доставляемыми агентом или энергией. Таким образом, доставка агента или энергии контролирует функцию органа.
EA202190358A 2018-07-31 2019-07-31 Способы генной терапии для контроля функции органов EA202190358A1 (ru)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
EA202190358A1 true EA202190358A1 (ru) 2021-05-04

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US (1) US20210301306A1 (ru)
EP (1) EP3829718A4 (ru)
JP (2) JP2021533126A (ru)
KR (1) KR20210052450A (ru)
CN (1) CN113301956A (ru)
AU (1) AU2019315445A1 (ru)
BR (1) BR112021001878A2 (ru)
CA (1) CA3108324A1 (ru)
EA (1) EA202190358A1 (ru)
IL (1) IL280531A (ru)
MX (1) MX2021001336A (ru)
PH (1) PH12021550243A1 (ru)
SG (1) SG11202101042SA (ru)
WO (1) WO2020028466A1 (ru)

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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

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4863457A (en) 1986-11-24 1989-09-05 Lee David A Drug delivery device
US5378475A (en) 1991-02-21 1995-01-03 University Of Kentucky Research Foundation Sustained release drug delivery devices
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
US5464758A (en) 1993-06-14 1995-11-07 Gossen; Manfred Tight control of gene expression in eucaryotic cells by tetracycline-responsive promoters
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
US6342390B1 (en) 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
FR2814642B1 (fr) 2000-10-03 2005-07-01 Ass Pour Le Dev De La Rech En Souris transgenique pour la recombinaison ciblee mediee par la cre-er modifiee
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
US20050287559A1 (en) * 2004-05-07 2005-12-29 Applera Corporation Genetic polymorphisms associated with vascular, methods of detection and uses thereof
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
US10711270B2 (en) * 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
CN108348528A (zh) * 2015-09-17 2018-07-31 科达生物治疗公司 治疗神经障碍的组合物和方法
US11771778B2 (en) * 2016-03-09 2023-10-03 Fadi ASSAF Using DREADD for neuronal modulation in treating neuronal diseases
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
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
WO2021081201A1 (en) * 2019-10-22 2021-04-29 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders

Also Published As

Publication number Publication date
JP2021533126A (ja) 2021-12-02
AU2019315445A1 (en) 2021-03-18
MX2021001336A (es) 2021-09-10
EP3829718A1 (en) 2021-06-09
WO2020028466A1 (en) 2020-02-06
IL280531A (en) 2021-03-25
KR20210052450A (ko) 2021-05-10
JP2024041871A (ja) 2024-03-27
SG11202101042SA (en) 2021-02-25
US20210301306A1 (en) 2021-09-30
PH12021550243A1 (en) 2021-11-22
CN113301956A (zh) 2021-08-24
BR112021001878A2 (pt) 2021-04-27
CA3108324A1 (en) 2020-02-06
EP3829718A4 (en) 2022-06-22

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