KR20220022107A - 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 - Google Patents

유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 Download PDF

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KR20220022107A
KR20220022107A KR1020217018249A KR20217018249A KR20220022107A KR 20220022107 A KR20220022107 A KR 20220022107A KR 1020217018249 A KR1020217018249 A KR 1020217018249A KR 20217018249 A KR20217018249 A KR 20217018249A KR 20220022107 A KR20220022107 A KR 20220022107A
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aav
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gly
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티모시 제이. 밀러
리나스 파데기마스
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아베오나 테라퓨틱스 인코퍼레이티드
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    • A—HUMAN NECESSITIES
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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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    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
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    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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KR1020217018249A 2018-12-05 2019-12-04 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 Ceased KR20220022107A (ko)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862775871P 2018-12-05 2018-12-05
US62/775,871 2018-12-05
US201962801195P 2019-02-05 2019-02-05
US62/801,195 2019-02-05
US201962863126P 2019-06-18 2019-06-18
US62/863,126 2019-06-18
US201962914856P 2019-10-14 2019-10-14
US62/914,856 2019-10-14
PCT/US2019/064396 WO2020117898A1 (en) 2018-12-05 2019-12-04 Recombinant adeno-associated viral vector for gene delivery

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KR20220022107A true KR20220022107A (ko) 2022-02-24

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KR1020217018249A Ceased KR20220022107A (ko) 2018-12-05 2019-12-04 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터

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US (1) US12454701B2 (https=)
EP (1) EP3890786A4 (https=)
JP (2) JP7590968B2 (https=)
KR (1) KR20220022107A (https=)
CN (1) CN113423434A (https=)
AU (1) AU2019391042A1 (https=)
BR (1) BR112021009913A2 (https=)
CA (1) CA3121177A1 (https=)
IL (1) IL283546A (https=)
MX (1) MX2021006646A (https=)
WO (1) WO2020117898A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
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WO2023249475A1 (ko) * 2022-06-24 2023-12-28 연세대학교 산학협력단 망막색소상피에 특이적으로 작동하는 crispr/cas 복합체를 유효성분으로 포함하는 망막질환 치료용 약학조성물
WO2024117285A1 (ko) * 2022-11-28 2024-06-06 (주)지뉴인텍 Pde6b 유전자가 포함된 아데노바이러스 재조합 벡터를 유효성분으로 포함하는 유전성 망막질환 예방 또는 치료용 조성물

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