CA3034887A1 - Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof - Google Patents

Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof Download PDF

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Publication number
CA3034887A1
CA3034887A1 CA3034887A CA3034887A CA3034887A1 CA 3034887 A1 CA3034887 A1 CA 3034887A1 CA 3034887 A CA3034887 A CA 3034887A CA 3034887 A CA3034887 A CA 3034887A CA 3034887 A1 CA3034887 A1 CA 3034887A1
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week
cell
light
polypeptide
nucleic acid
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CA3034887A
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English (en)
French (fr)
Inventor
Zhuo-Hua Pan
Tushar Ganjawala
Qi Lu
Gary Abrams
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Wayne State University
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Wayne State University
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/405—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from algae
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0621—Eye cells, e.g. cornea, iris pigmented cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2510/00—Genetically modified cells
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3034887A 2016-08-29 2017-08-29 Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof Pending CA3034887A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662380871P 2016-08-29 2016-08-29
US62/380,871 2016-08-29
PCT/US2017/049158 WO2018044912A1 (en) 2016-08-29 2017-08-29 Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof

Publications (1)

Publication Number Publication Date
CA3034887A1 true CA3034887A1 (en) 2018-03-08

Family

ID=59859616

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3034887A Pending CA3034887A1 (en) 2016-08-29 2017-08-29 Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof

Country Status (15)

Country Link
US (3) US11041004B2 (https=)
EP (1) EP3504227A1 (https=)
JP (1) JP7116884B2 (https=)
KR (3) KR102609571B1 (https=)
CN (2) CN110023327B (https=)
AU (4) AU2017319306B2 (https=)
BR (1) BR112019003950A2 (https=)
CA (1) CA3034887A1 (https=)
IL (2) IL265010B2 (https=)
MX (1) MX2019002321A (https=)
MY (1) MY197491A (https=)
NZ (1) NZ751983A (https=)
RU (1) RU2019109021A (https=)
SG (2) SG10202102061SA (https=)
WO (1) WO2018044912A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3504227A1 (en) 2016-08-29 2019-07-03 Wayne State University Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof
CN113173984B (zh) 2021-04-12 2022-08-30 中眸医疗科技(武汉)有限公司 一种新型光敏感通道蛋白vr1.0在制备视网膜感光细胞退行性疾病药物中的应用
MX2024007770A (es) * 2021-12-20 2024-07-01 Genans Biotechnology Co Ltd Restauracion visual optogenetica utilizando neuropsina acoplada a gq sensible a la luz (opsin 5).
EP4665750A1 (en) 2023-02-17 2025-12-24 Ray Therapeutics, Inc. Gene therapy compositions and methods for treating diseases of the retina
WO2025137649A1 (en) * 2023-12-22 2025-06-26 Wayne State University Method of enhancing viral mediated gene delivery

Family Cites Families (21)

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Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
IE913929A1 (en) 1990-11-13 1992-05-20 Immunex Corp Bifunctional selectable fusion genes
AU6953394A (en) 1993-05-21 1994-12-20 Targeted Genetics Corporation Bifunctional selectable fusion genes based on the cytosine deaminase (cd) gene
EP0745134A1 (en) 1994-02-22 1996-12-04 Danafarber Cancer Institute Nucleic acid delivery system, method of synthesis and uses thereof
EP1919497B1 (en) 2005-07-22 2020-02-12 The Board of Trustees of the Leland Stanford Junior University Light-activated cation channel and uses thereof
CN101484005A (zh) 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
KR100922422B1 (ko) 2006-06-27 2009-10-16 가부시키가이샤 어드밴테스트 반도체 시험 장치 및 반도체 메모리의 시험 방법
WO2011140279A1 (en) 2010-05-04 2011-11-10 Wayne State University Aav-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells
KR101945962B1 (ko) 2010-09-08 2019-02-08 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. 돌연변이 채널로돕신 2
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
WO2013071231A1 (en) * 2011-11-12 2013-05-16 Massachusetts Institute Of Technology Channelrhodopsins for optical control of cells
CA2866405A1 (en) * 2012-03-05 2013-09-12 Wayne State University Identification of channelopsin-2 (chop2) mutations and methods of use
EP2968476B1 (en) * 2013-03-14 2021-05-05 Wayne State University A plasmin for use in enhancing delivery of therapeutic compounds to the eyes or for improving vision
EP3102290A1 (en) * 2014-02-07 2016-12-14 Massachusetts Institute Of Technology Blue light-activated ion channel molecules and uses thereof
EP4678754A3 (en) 2014-03-11 2026-04-08 Wayne State University A modified mglur6 promoter and methods of use
WO2015161308A1 (en) 2014-04-18 2015-10-22 Massachusetts Institute Of Technology Mutant channelrhodopsins with altered ion selectivity
WO2016179038A1 (en) 2015-05-01 2016-11-10 Spark Therapeutics, Inc. ADENO-ASSOCIATED VIRUS-MEDIATED CRISPR-Cas9 TREATMENT OF OCULAR DISEASE
LT3436051T (lt) 2016-03-31 2021-11-25 Spark Therapeutics, Inc. Kolonėlėmis pagrįstas visiškai keičiamo masto raav gavimo būdas
WO2017210664A1 (en) 2016-06-03 2017-12-07 Massachusetts Institute Of Technology Somatic opsins for single cell resolution optogenetics
AU2017302013B2 (en) 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP3504227A1 (en) 2016-08-29 2019-07-03 Wayne State University Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof

Also Published As

Publication number Publication date
IL265010B1 (en) 2024-02-01
SG11201901697QA (en) 2019-03-28
JP7116884B2 (ja) 2022-08-12
US11041004B2 (en) 2021-06-22
RU2019109021A (ru) 2020-09-29
WO2018044912A1 (en) 2018-03-08
KR102609571B9 (ko) 2025-11-20
IL310142A (en) 2024-03-01
EP3504227A1 (en) 2019-07-03
JP2019528781A (ja) 2019-10-17
AU2024200016B2 (en) 2025-07-17
AU2024200016A1 (en) 2024-01-25
BR112019003950A2 (pt) 2019-06-25
KR20250159068A (ko) 2025-11-07
US20250163111A1 (en) 2025-05-22
US12234267B2 (en) 2025-02-25
KR20230169427A (ko) 2023-12-15
NZ751983A (en) 2025-11-28
AU2022201291B2 (en) 2023-10-05
MY197491A (en) 2023-06-19
IL265010A (https=) 2019-04-30
AU2024200016B9 (en) 2025-08-07
KR20190075906A (ko) 2019-07-01
KR102609571B1 (ko) 2023-12-01
AU2017319306B2 (en) 2021-12-02
AU2022201291A1 (en) 2022-03-17
CN119979612A (zh) 2025-05-13
MX2019002321A (es) 2019-10-21
US20220089660A1 (en) 2022-03-24
SG10202102061SA (en) 2021-04-29
US20190241628A1 (en) 2019-08-08
RU2019109021A3 (https=) 2021-02-02
AU2025248765A1 (en) 2025-10-30
AU2017319306A1 (en) 2019-04-11
IL265010B2 (en) 2024-06-01
KR102877327B1 (ko) 2025-10-27
CN110023327B (zh) 2024-02-27
CN110023327A (zh) 2019-07-16

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