KR20220157944A - 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법 - Google Patents

인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법 Download PDF

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KR20220157944A
KR20220157944A KR1020227030282A KR20227030282A KR20220157944A KR 20220157944 A KR20220157944 A KR 20220157944A KR 1020227030282 A KR1020227030282 A KR 1020227030282A KR 20227030282 A KR20227030282 A KR 20227030282A KR 20220157944 A KR20220157944 A KR 20220157944A
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amino acids
acid substitutions
amino acid
amino
acids
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Korean (ko)
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엠마누엘 존 시몬스
로버트 엔지
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아카우오스, 인크.
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    • 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
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/103—Ovine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
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    • 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
    • C12N2750/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020227030282A 2020-02-21 2021-02-19 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법 Pending KR20220157944A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062979792P 2020-02-21 2020-02-21
US62/979,792 2020-02-21
PCT/US2021/018919 WO2021168362A1 (en) 2020-02-21 2021-02-19 Compositions and methods for treating non-age-associated hearing impairment in a human subject

Publications (1)

Publication Number Publication Date
KR20220157944A true KR20220157944A (ko) 2022-11-29

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KR1020227030282A Pending KR20220157944A (ko) 2020-02-21 2021-02-19 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법

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Country Link
US (4) US11807867B2 (https=)
EP (1) EP4114958A1 (https=)
JP (1) JP7807380B2 (https=)
KR (1) KR20220157944A (https=)
CN (1) CN116096905A (https=)
AR (1) AR121390A1 (https=)
AU (1) AU2021225035A1 (https=)
BR (1) BR112022016596A2 (https=)
CA (1) CA3168055A1 (https=)
CL (1) CL2022002276A1 (https=)
IL (1) IL295747A (https=)
MX (1) MX2022010050A (https=)
TW (1) TWI886216B (https=)
WO (1) WO2021168362A1 (https=)

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CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
JP7807380B2 (ja) 2020-02-21 2026-01-27 アコーオス インコーポレイテッド ヒト対象において非加齢関連聴力障害を処置するための組成物および方法
AU2021247172A1 (en) 2020-04-01 2022-09-15 University Of Florida Research Foundation, Incorporated Dual AAV-MY07A vectors with improved safety for the treatment of USH1B
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CN117305367A (zh) * 2023-08-21 2023-12-29 复旦大学附属眼耳鼻喉科医院 一种表达全长耳畸蛋白的双aav载体系统及其应用
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