CA2999279A1 - Targeted expression of chloride channels and methods of use thereof - Google Patents

Targeted expression of chloride channels and methods of use thereof Download PDF

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Publication number
CA2999279A1
CA2999279A1 CA2999279A CA2999279A CA2999279A1 CA 2999279 A1 CA2999279 A1 CA 2999279A1 CA 2999279 A CA2999279 A CA 2999279A CA 2999279 A CA2999279 A CA 2999279A CA 2999279 A1 CA2999279 A1 CA 2999279A1
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CA
Canada
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.)
Abandoned
Application number
CA2999279A
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English (en)
French (fr)
Inventor
Griffith Roger Thomas
Shawnalea Jimee FRAZIER
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Goleini Inc
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Goleini Inc
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 Goleini Inc filed Critical Goleini Inc
Publication of CA2999279A1 publication Critical patent/CA2999279A1/en
Abandoned legal-status Critical Current

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    • 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)
  • 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)
CA2999279A 2015-10-01 2016-09-28 Targeted expression of chloride channels and methods of use thereof Abandoned CA2999279A1 (en)

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

Publications (1)

Publication Number Publication Date
CA2999279A1 true CA2999279A1 (en) 2017-04-06

Family

ID=58427328

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2999279A Abandoned CA2999279A1 (en) 2015-10-01 2016-09-28 Targeted expression of chloride channels and methods of use thereof

Country Status (11)

Country Link
US (1) US11407812B2 (https=)
EP (1) EP3355938B1 (https=)
JP (1) JP7097070B2 (https=)
KR (2) KR20180072713A (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
SG11202105079QA (en) * 2018-11-14 2021-06-29 Flagship Pioneering Innovations V Inc Fusosome compositions for cns delivery
US12496274B2 (en) 2018-11-14 2025-12-16 Flagship Pioneering Innovations V, Inc. Compositions and methods for compartment-specific cargo 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
EP1767637A1 (en) * 2000-06-20 2007-03-28 Bionomics Limited Mutation associated with epilepsy
AU2002250076A1 (en) * 2001-02-16 2002-09-04 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
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
WO2009133191A1 (en) * 2008-04-30 2009-11-05 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
WO2011137210A2 (en) 2010-04-30 2011-11-03 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
KR102530118B1 (ko) * 2012-07-25 2023-05-08 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
US9624288B2 (en) 2012-12-10 2017-04-18 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
US20150283397A1 (en) 2014-03-27 2015-10-08 Circuit Therapeutics, Inc. System and method for therapeutic management of cough
CN107002096A (zh) 2014-10-06 2017-08-01 阿罗根有限公司 基于aav的基因疗法
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
KR20230079511A (ko) 2016-04-21 2023-06-07 바이로베크 인코포레이티드 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물

Also Published As

Publication number Publication date
BR112018006480A2 (pt) 2018-10-09
EP3355938A4 (en) 2019-03-06
US20190010210A1 (en) 2019-01-10
AU2016332847A2 (en) 2023-08-03
HK1252915A1 (zh) 2019-06-06
US11407812B2 (en) 2022-08-09
JP2018533976A (ja) 2018-11-22
WO2017058926A1 (en) 2017-04-06
JP7097070B2 (ja) 2022-07-07
AU2016332847B2 (en) 2023-08-10
AU2016332847A1 (en) 2018-04-19
CN108289965A (zh) 2018-07-17
EP3355938B1 (en) 2024-02-07
KR20250012721A (ko) 2025-01-24
KR20180072713A (ko) 2018-06-29
SG10201912703PA (en) 2020-02-27
EP3355938A1 (en) 2018-08-08

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Effective date: 20210922

FZDE Discontinued

Effective date: 20240501