KR20250012721A - 클로라이드 채널의 표적된 발현 및 이의 사용 방법 - Google Patents

클로라이드 채널의 표적된 발현 및 이의 사용 방법 Download PDF

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KR20250012721A
KR20250012721A KR1020257000627A KR20257000627A KR20250012721A KR 20250012721 A KR20250012721 A KR 20250012721A KR 1020257000627 A KR1020257000627 A KR 1020257000627A KR 20257000627 A KR20257000627 A KR 20257000627A KR 20250012721 A KR20250012721 A KR 20250012721A
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subunit
vector
cells
promoter
certain embodiments
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Korean (ko)
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그리피스 로저 토마스
샤날리 지미 프레이저
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고레이니 인크.
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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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
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • 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
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/16011Herpesviridae
    • C12N2710/16111Cytomegalovirus, e.g. human herpesvirus 5
    • C12N2710/16141Use of virus, viral particle or viral elements as a vector
    • C12N2710/16143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
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  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020257000627A 2015-10-01 2016-09-28 클로라이드 채널의 표적된 발현 및 이의 사용 방법 Pending KR20250012721A (ko)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US201562235920P 2015-10-01 2015-10-01
US201562235914P 2015-10-01 2015-10-01
US62/235,920 2015-10-01
US62/235,914 2015-10-01
US201662303907P 2016-03-04 2016-03-04
US62/303,907 2016-03-04
US201662378509P 2016-08-23 2016-08-23
US62/378,509 2016-08-23
PCT/US2016/054199 WO2017058926A1 (en) 2015-10-01 2016-09-28 Targeted expression of chloride channels and methods of use thereof
KR1020187012245A KR20180072713A (ko) 2015-10-01 2016-09-28 클로라이드 채널의 표적된 발현 및 이의 사용 방법

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020187012245A Division KR20180072713A (ko) 2015-10-01 2016-09-28 클로라이드 채널의 표적된 발현 및 이의 사용 방법

Publications (1)

Publication Number Publication Date
KR20250012721A true KR20250012721A (ko) 2025-01-24

Family

ID=58427328

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020257000627A Pending KR20250012721A (ko) 2015-10-01 2016-09-28 클로라이드 채널의 표적된 발현 및 이의 사용 방법
KR1020187012245A Ceased KR20180072713A (ko) 2015-10-01 2016-09-28 클로라이드 채널의 표적된 발현 및 이의 사용 방법

Family Applications After (1)

Application Number Title Priority Date Filing Date
KR1020187012245A Ceased KR20180072713A (ko) 2015-10-01 2016-09-28 클로라이드 채널의 표적된 발현 및 이의 사용 방법

Country Status (11)

Country Link
US (1) US11407812B2 (https=)
EP (1) EP3355938B1 (https=)
JP (1) JP7097070B2 (https=)
KR (2) KR20250012721A (https=)
CN (1) CN108289965A (https=)
AU (1) AU2016332847B2 (https=)
BR (1) BR112018006480A2 (https=)
CA (1) CA2999279A1 (https=)
HK (1) HK1252915A1 (https=)
SG (1) SG10201912703PA (https=)
WO (1) WO2017058926A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220008557A1 (en) * 2018-11-14 2022-01-13 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
CN113631718A (zh) 2018-11-14 2021-11-09 旗舰先锋创新V股份有限公司 用于特定隔室货物递送的组合物和方法
CN110679548B (zh) * 2019-10-24 2021-11-30 南方医科大学南方医院 一种自闭症小鼠模型的构建方法
CN111759857A (zh) * 2020-06-30 2020-10-13 宁波市微循环与莨菪类药研究所 Gabrd甲基化靶点在制备抗海洛因复吸药物中的应用
CN115480485B (zh) * 2022-09-14 2024-09-10 南开大学 一种基于数据驱动模型的软镜扭转运动安全控制方法
GB202401492D0 (en) 2024-02-05 2024-03-20 Ucl Business Ltd Chloride channels and uses thereof
CN119331917B (zh) * 2024-10-28 2025-10-17 北京爱思益普生物科技股份有限公司 能够表达GlyR α1/β型GlyR的细胞株及其制备方法

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3644643A (en) * 1969-10-13 1972-02-22 Unimed Inc Method of reducing intraocular pressure using glycine
US4559157A (en) 1983-04-21 1985-12-17 Creative Products Resource Associates, Ltd. Cosmetic applicator useful for skin moisturizing
LU84979A1 (fr) 1983-08-30 1985-04-24 Oreal Composition cosmetique ou pharmaceutique sous forme aqueuse ou anhydre dont la phase grasse contient un polyether oligomere et polyethers oligomeres nouveaux
US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
US4820508A (en) 1987-06-23 1989-04-11 Neutrogena Corporation Skin protective composition
US4992478A (en) 1988-04-04 1991-02-12 Warner-Lambert Company Antiinflammatory skin moisturizing composition and method of preparing same
US5527703A (en) * 1994-05-25 1996-06-18 Merck & Co., Inc. DNA encoding glutamate gated chloride channels
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6464986B1 (en) * 2000-04-14 2002-10-15 Allegan Sales, Inc. Method for treating pain by peripheral administration of a neurotoxin
ATE437948T1 (de) 2000-06-20 2009-08-15 Bionomics Ltd Mutation, welche mit epilepsie assoziiert ist
CA2438386A1 (en) * 2001-02-16 2002-08-29 Bristol-Myers Squibb Company Polynucleotides encoding a novel glycine receptor alpha subunit expressed in the gastrointestinal tract, hgra4, and splice variant thereof
US20040058313A1 (en) 2002-04-24 2004-03-25 Abreu Marcio Marc Compositions, targets, methods and devices for the therapy of ocular and periocular disorders
WO2007127428A2 (en) * 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
WO2008024998A2 (en) 2006-08-24 2008-02-28 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
WO2008143875A1 (en) * 2007-05-14 2008-11-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Targeted delivery of glycine receptors to excitable cells
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
EP2283038A1 (en) 2008-04-30 2011-02-16 Dublin City University Compositions and methods for expressing in-frame multimeric proteins
EP2344636B1 (en) * 2008-10-09 2017-12-06 Howard Hughes Medical Institute Novel chimeric ligand-gated ion channels and methods of use thereof
US20130184318A1 (en) 2010-04-30 2013-07-18 The Regents Of The University Of California Modulating compliance of trabecular meshwork
WO2012155091A1 (en) 2011-05-11 2012-11-15 Kirax Corporation Package for improved treatment of conditions
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
EP2692868A1 (en) * 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
WO2014093251A1 (en) * 2012-12-10 2014-06-19 University Of Pittsburgh Of The Commonwealth System Of Higher Education Engineered receptors and their use
AU2015204691A1 (en) 2014-01-08 2016-07-21 Circuit Therapeutics, Inc. Method for therapeutic management of ocular hypertension
US20150283398A1 (en) 2014-03-27 2015-10-08 Circuit Therapeutics, Inc. System and method for therapeutic management of cough
EP3204503A1 (en) 2014-10-06 2017-08-16 Arthrogen BV Aav-based gene therapy
AU2016324317A1 (en) * 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
GB2547179A (en) 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
JP7496667B2 (ja) 2016-04-21 2024-06-07 ビロベク,インコーポレイテッド 昆虫細胞中でのaav生成、方法およびその組成物

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Publication number Publication date
AU2016332847B2 (en) 2023-08-10
US11407812B2 (en) 2022-08-09
AU2016332847A2 (en) 2023-08-03
WO2017058926A1 (en) 2017-04-06
JP7097070B2 (ja) 2022-07-07
EP3355938B1 (en) 2024-02-07
KR20180072713A (ko) 2018-06-29
CN108289965A (zh) 2018-07-17
BR112018006480A2 (pt) 2018-10-09
EP3355938A1 (en) 2018-08-08
CA2999279A1 (en) 2017-04-06
HK1252915A1 (zh) 2019-06-06
JP2018533976A (ja) 2018-11-22
US20190010210A1 (en) 2019-01-10
EP3355938A4 (en) 2019-03-06
AU2016332847A1 (en) 2018-04-19
SG10201912703PA (en) 2020-02-27

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