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

    • 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
    • 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
    • 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]
    • 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
    • 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
    • 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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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

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)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
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
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

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 (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3880179A2 (en) * 2018-11-14 2021-09-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
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
DE60139398D1 (de) * 2000-06-20 2009-09-10 Bionomics Ltd Mutation, welche mit epilepsie assoziiert ist
WO2002066606A2 (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
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
DK2061891T3 (da) 2006-08-24 2012-07-23 Virovek Inc Ekspression i insektceller af gener med overlappende åbne læserammer, fremgangsmåder og sammensætninger hertil
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
JP5775819B2 (ja) * 2008-10-09 2015-09-09 ハワード ヒューズ メディカル インスティチュート 新規なキメラリガンド開口型イオンチャネルおよびその使用方法
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
EP2877213B1 (en) * 2012-07-25 2020-12-02 The Broad Institute, Inc. 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
WO2015106005A1 (en) 2014-01-08 2015-07-16 Circuit Therapeutics, Inc. Method for therapeutic management of ocular hypertension
AU2015237140A1 (en) 2014-03-27 2016-10-13 Circuit Therapeutics, Inc. System and method for therapeutic management of cough
JP2017531652A (ja) 2014-10-06 2017-10-26 アルスロジェン ビー.ブイ.Arthrogen B.V. Aavに基づく遺伝子治療
KR20200108514A (ko) 2015-09-17 2020-09-21 코다 바이오테라퓨틱스 인코포레이티드 신경 장애를 치료하기 위한 조성물 및 방법
GB2547179A (en) 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
KR20180134846A (ko) 2016-04-21 2018-12-19 바이로베크 인코포레이티드 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물

Also Published As

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

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
JP7766393B2 (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
CN116406304A (zh) 使用AIMP2-DX2和任选地miR-142的靶序列及其组合物治疗年龄相关的黄斑疾病的方法
EP4165193A2 (en) Aav-mediated gene transfer for retinopathy
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
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