KR102528641B1 - Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 - Google Patents

Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 Download PDF

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KR102528641B1
KR102528641B1 KR1020207025137A KR20207025137A KR102528641B1 KR 102528641 B1 KR102528641 B1 KR 102528641B1 KR 1020207025137 A KR1020207025137 A KR 1020207025137A KR 20207025137 A KR20207025137 A KR 20207025137A KR 102528641 B1 KR102528641 B1 KR 102528641B1
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앨버트 반즈 세이무어
씨민 세어 아메드
제이슨 보크 라이트
세레나 니콜 돌리브
힐러드 루빈
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호몰로지 메디슨, 인크.
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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KR1020207025137A 2018-02-01 2019-02-01 Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 Active KR102528641B1 (ko)

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KR1020237014637A KR20230065367A (ko) 2018-02-01 2019-02-01 Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법

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US201862625150P 2018-02-01 2018-02-01
US62/625,150 2018-02-01
PCT/US2019/016351 WO2019152841A1 (en) 2018-02-01 2019-02-01 Adeno-associated virus compositions for pah gene transfer and methods of use thereof

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KR1020237014637A Division KR20230065367A (ko) 2018-02-01 2019-02-01 Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법

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KR102528641B1 true KR102528641B1 (ko) 2023-05-03

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KR1020237014637A Ceased KR20230065367A (ko) 2018-02-01 2019-02-01 Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법

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PE20190401A1 (es) 2016-07-26 2019-03-13 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado
BR112020022722A8 (pt) 2018-05-09 2022-01-18 Biomarin Pharm Inc Genoma e seu uso, vetor, partícula, molécula de ácido nucleico isolada, ácido nucleico, composição, tratamento, método
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
EP3980447A4 (en) * 2019-06-10 2023-07-26 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR ARSA GENE TRANSFER AND METHODS OF USE THEREOF
TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
IL294713A (en) * 2020-01-16 2022-09-01 Takeda Pharmaceuticals Co Adeno associated virus based gene therapy for phenylketonuria
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
CN116656740A (zh) * 2023-05-30 2023-08-29 浙江大学 一种腺相关病毒基因治疗载体及其在制备治疗苯丙酮尿症的药物中的应用
CN119876192B (zh) * 2024-07-02 2025-11-07 浙江大学滨江研究院 用于治疗苯丙酮尿症的多核苷酸

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WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
US20170211094A1 (en) 2010-04-30 2017-07-27 City Of Hope Recombinant adeno-associated vectors for targeted treatment

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AU2003237159A1 (en) * 2002-04-30 2003-11-17 University Of Florida Treatment for phenylketonuria
WO2010124180A1 (en) * 2009-04-23 2010-10-28 Phasebio Pharmaceuticals, Inc. Phenylalanine hydroxylase fusion protein and methods for treating phenylketonuria
DK2675902T3 (da) * 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
EA034103B1 (ru) * 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료

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US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
US20170211094A1 (en) 2010-04-30 2017-07-27 City Of Hope Recombinant adeno-associated vectors for targeted treatment
WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof

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EP3746113A1 (en) 2020-12-09
JP2022105105A (ja) 2022-07-12
WO2019152841A1 (en) 2019-08-08
IL276215B (en) 2022-10-01
JP7066861B2 (ja) 2022-05-13
JP2021512601A (ja) 2021-05-20
JP7433360B2 (ja) 2024-02-19
KR20200127187A (ko) 2020-11-10
IL276215B2 (en) 2023-02-01
CA3089824A1 (en) 2019-08-08
MX2020008085A (es) 2020-12-10
IL295830A (en) 2022-10-01
BR112020015511A2 (pt) 2021-01-26
PE20210341A1 (es) 2021-02-23
EP3746113A4 (en) 2022-03-09
KR20230065367A (ko) 2023-05-11
IL276215A (en) 2020-09-30
AU2019215150A1 (en) 2020-09-24
CN111886021A (zh) 2020-11-03
CL2020001992A1 (es) 2021-02-12
EA202091844A1 (ru) 2021-02-03
CO2020010783A2 (es) 2020-11-20

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