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
Authority
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
Other languages
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
Original Assignee
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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    • 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)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (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)
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
US62/235,920 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

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 (OSRAM)
EP (1) EP3355938B1 (OSRAM)
JP (1) JP7097070B2 (OSRAM)
KR (2) KR20250012721A (OSRAM)
CN (1) CN108289965A (OSRAM)
AU (1) AU2016332847B2 (OSRAM)
BR (1) BR112018006480A2 (OSRAM)
CA (1) CA2999279A1 (OSRAM)
HK (1) HK1252915A1 (OSRAM)
SG (1) SG10201912703PA (OSRAM)
WO (1) WO2017058926A1 (OSRAM)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019378036B2 (en) 2018-11-14 2026-05-07 Flagship Pioneering Innovations V, Inc Compositions and methods for compartment-specific cargo delivery
CA3120082A1 (en) * 2018-11-14 2020-05-22 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
CN114214396A (zh) * 2020-06-30 2022-03-22 宁波市康宁医院(宁波市精神疾病预防控制中心、宁波市微循环与莨菪类药研究所) Gabrd甲基化作为抗海洛因复吸靶点的应用
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

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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
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Also Published As

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

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Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20210922

FZDE Discontinued

Effective date: 20240501