KR20180072713A - 클로라이드 채널의 표적된 발현 및 이의 사용 방법 - Google Patents

클로라이드 채널의 표적된 발현 및 이의 사용 방법 Download PDF

Info

Publication number
KR20180072713A
KR20180072713A KR1020187012245A KR20187012245A KR20180072713A KR 20180072713 A KR20180072713 A KR 20180072713A KR 1020187012245 A KR1020187012245 A KR 1020187012245A KR 20187012245 A KR20187012245 A KR 20187012245A KR 20180072713 A KR20180072713 A KR 20180072713A
Authority
KR
South Korea
Prior art keywords
vector
subunit
promoter
glyr
nucleic acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
KR1020187012245A
Other languages
English (en)
Korean (ko)
Inventor
그리피스 로저 토마스
샤날리 지미 프레이저
Original Assignee
고레이니 인크.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 고레이니 인크. filed Critical 고레이니 인크.
Priority to KR1020257000627A priority Critical patent/KR20250012721A/ko
Publication of KR20180072713A publication Critical patent/KR20180072713A/ko
Ceased legal-status Critical Current

Links

Images

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • 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
    • 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
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
    • C12N2710/16141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • Y—GENERAL 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
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (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)
KR1020187012245A 2015-10-01 2016-09-28 클로라이드 채널의 표적된 발현 및 이의 사용 방법 Ceased KR20180072713A (ko)

Priority Applications (1)

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

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201562235914P 2015-10-01 2015-10-01
US201562235920P 2015-10-01 2015-10-01
US62/235,914 2015-10-01
US62/235,920 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

Related Child Applications (1)

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

Publications (1)

Publication Number Publication Date
KR20180072713A true KR20180072713A (ko) 2018-06-29

Family

ID=58427328

Family Applications (2)

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
KR1020257000627A Pending KR20250012721A (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
KR1020257000627A Pending KR20250012721A (ko) 2015-10-01 2016-09-28 클로라이드 채널의 표적된 발현 및 이의 사용 방법

Country Status (11)

Country Link
US (1) US11407812B2 (enExample)
EP (1) EP3355938B1 (enExample)
JP (1) JP7097070B2 (enExample)
KR (2) KR20180072713A (enExample)
CN (1) CN108289965A (enExample)
AU (1) AU2016332847B2 (enExample)
BR (1) BR112018006480A2 (enExample)
CA (1) CA2999279A1 (enExample)
HK (1) HK1252915A1 (enExample)
SG (1) SG10201912703PA (enExample)
WO (1) WO2017058926A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019378881A1 (en) * 2018-11-14 2021-06-03 Flagship Pioneering Innovations V, Inc. Fusosome compositions for CNS delivery
JP2022513040A (ja) 2018-11-14 2022-02-07 フラッグシップ パイオニアリング イノベーションズ ブイ， インコーポレイテッド コンパートメント特異的カーゴ送達のための組成物および方法
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
EP1292676B1 (en) * 2000-06-20 2009-07-29 Bionomics Limited Mutation associated with epilepsy
US7056737B2 (en) * 2001-02-16 2006-06-06 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
US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
US20080289058A1 (en) 2007-05-14 2008-11-20 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
WO2009133191A1 (en) * 2008-04-30 2009-11-05 Dublin City University Compositions and methods for expressing in-frame multimeric proteins
US8435762B2 (en) 2008-10-09 2013-05-07 Howard Hughes Medical Institute Chimeric ligand-gated ion channels and methods of use thereof
EP2563929A4 (en) 2010-04-30 2014-05-14 Univ California MODULATION OF THE INTAKTHEIS OF THE TRAVERKELWERKS
TW201306825A (zh) * 2011-05-11 2013-02-16 Kirax Corp 狀況治療之改良套裝
KR20230065381A (ko) 2012-07-25 2023-05-11 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
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
JP2017501834A (ja) 2014-01-08 2017-01-19 サーキット セラピューティクス， インコーポレイテッド 高眼圧症の治療管理のための方法
US20150283398A1 (en) 2014-03-27 2015-10-08 Circuit Therapeutics, Inc. System and method for therapeutic management of cough
CA2963168A1 (en) 2014-10-06 2016-04-14 Arthrogen B.V. Aav-based gene therapy
CA2998491A1 (en) * 2015-09-17 2017-03-23 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
GB2547179A (en) 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
AU2017254652B2 (en) 2016-04-21 2022-12-01 Virovek, Inc. AAV production in insect cells, methods and compositions therefor

Also Published As

Publication number Publication date
AU2016332847A2 (en) 2023-08-03
JP2018533976A (ja) 2018-11-22
SG10201912703PA (en) 2020-02-27
AU2016332847A1 (en) 2018-04-19
EP3355938B1 (en) 2024-02-07
WO2017058926A1 (en) 2017-04-06
EP3355938A1 (en) 2018-08-08
CN108289965A (zh) 2018-07-17
AU2016332847B2 (en) 2023-08-10
BR112018006480A2 (pt) 2018-10-09
US11407812B2 (en) 2022-08-09
US20190010210A1 (en) 2019-01-10
JP7097070B2 (ja) 2022-07-07
CA2999279A1 (en) 2017-04-06
KR20250012721A (ko) 2025-01-24
HK1252915A1 (zh) 2019-06-06
EP3355938A4 (en) 2019-03-06

Similar Documents

Publication Publication Date Title
KR20180072713A (ko) 클로라이드 채널의 표적된 발현 및 이의 사용 방법
JP7097398B2 (ja) ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター
JP7057281B2 (ja) 眼疾患のための遺伝子療法
US11617801B2 (en) RP2 and RPGR vectors for treating X-linked retinitis pigmentosa
JP2020527335A (ja) 眼疾患のための遺伝子療法
WO2023116745A1 (zh) 优化的cyp4v2基因及其用途
EP3622958B1 (en) Use of potassium ion channel inhibitor for treatment of depression and pharmaceutical composition
EP4165193A2 (en) Aav-mediated gene transfer for retinopathy
EP4695613A2 (en) Methods of modifying neurons in vivo to treat and/or prevent amyotrophic lateral sclerosis (als)
JP2025508336A (ja) Ｔｐｋを発現する組換えウイルスおよびアルツハイマー病の処置におけるその使用
HK40066183B (zh) 靶向akt通路的神经保护性基因疗法
HK40066183A (zh) 靶向akt通路的神经保护性基因疗法
Wan et al. AAV-DJ–Mediated MYOC Silencing as a Gene Therapy Approach for Myocilin-Associated Glaucoma
WO2022263845A1 (en) Ocular disorders
KR20220152550A (ko) Nmnat1-연관 망막 변성의 유전자 치료법

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20180430

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20210927

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20240131

Patent event code: PE09021S01D

E601 Decision to refuse application
PE0601 Decision on rejection of patent

Patent event date: 20240809

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D