IL301057A - Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof - Google Patents
Recombinant adeno associated virus (raav) encoding gjb2 and uses thereofInfo
- Publication number
- IL301057A IL301057A IL301057A IL30105723A IL301057A IL 301057 A IL301057 A IL 301057A IL 301057 A IL301057 A IL 301057A IL 30105723 A IL30105723 A IL 30105723A IL 301057 A IL301057 A IL 301057A
- Authority
- IL
- Israel
- Prior art keywords
- gjb2
- nucleic acid
- isolated nucleic
- seq
- cells
- Prior art date
Links
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- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
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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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- 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
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- 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
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- 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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- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
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- 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
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- 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
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063078233P | 2020-09-14 | 2020-09-14 | |
| US202163161619P | 2021-03-16 | 2021-03-16 | |
| PCT/US2021/050205 WO2022056444A1 (en) | 2020-09-14 | 2021-09-14 | Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof |
Publications (1)
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|---|---|
| IL301057A true IL301057A (en) | 2023-05-01 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL301057A IL301057A (en) | 2020-09-14 | 2021-09-14 | Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof |
Country Status (10)
| Country | Link |
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| WO2021067448A1 (en) * | 2019-09-30 | 2021-04-08 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| WO2024011224A2 (en) * | 2022-07-08 | 2024-01-11 | The Trustees Of Columbia University In The City Of New York | Regulatory element for cell type specific expression of genes in spinal motor neurons |
| WO2024218192A1 (en) * | 2023-04-18 | 2024-10-24 | Uniqure Biopharma B.V. | Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs |
| EP4713463A1 (en) * | 2023-05-18 | 2026-03-25 | Decibel Therapeutics, Inc. | Compositions and methods for treating gjb2-related hearing loss |
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| EP1308458A1 (en) * | 2001-10-30 | 2003-05-07 | GenOdyssee | New polynucleotides and polypeptides of the CX26 gene |
| DE602005019174D1 (de) * | 2004-06-17 | 2010-03-18 | Epigenomics Ag | Zusammensetzungen und verfahren zur verhinderung des mitschleppens einer kontamination in nukleinsäure-amplifikationsreaktionen |
| CN101389769A (zh) * | 2005-12-28 | 2009-03-18 | 森托科尔公司 | 用于评价和治疗银屑病及相关疾病的标记与方法 |
| WO2019200016A1 (en) * | 2018-04-10 | 2019-10-17 | President And Fellows Of Harvard College | Aav vectors encoding clarin-1 or gjb2 and uses thereof |
| WO2020077295A1 (en) * | 2018-10-11 | 2020-04-16 | Decibel Therapeutics, Inc. | Aav1 vectors and uses thereof for treatment of otic indications |
| EP4063510A1 (en) * | 2018-11-07 | 2022-09-28 | Akouos, Inc. | Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear |
| WO2021067448A1 (en) * | 2019-09-30 | 2021-04-08 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| WO2021231808A2 (en) * | 2020-05-13 | 2021-11-18 | Akouos, Inc. | Compositions and methods for treating gjb2-associated hearing loss |
| MX2023013394A (es) * | 2021-05-13 | 2023-12-14 | Akouos Inc | Composiciones de suministro de terapia genica y metodos para tratar perdida auditiva. |
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| WO2022056444A1 (en) | 2022-03-17 |
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