CN116157527A - 用于溶酶体病症的基因疗法 - Google Patents

用于溶酶体病症的基因疗法 Download PDF

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Publication number
CN116157527A
CN116157527A CN202180062323.1A CN202180062323A CN116157527A CN 116157527 A CN116157527 A CN 116157527A CN 202180062323 A CN202180062323 A CN 202180062323A CN 116157527 A CN116157527 A CN 116157527A
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raav
subject
dose
day
sirolimus
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Chinese (zh)
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阿萨·阿贝利奥维奇
杰弗里·思维格尼
特拉维斯·刘易斯
奥尔加·乌斯片斯卡亚
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Privelle Therapeutics
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Privelle Therapeutics
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CN202180062323.1A 2020-08-10 2021-08-10 用于溶酶体病症的基因疗法 Pending CN116157527A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063063851P 2020-08-10 2020-08-10
US63/063,851 2020-08-10
PCT/US2021/045447 WO2022035903A2 (en) 2020-08-10 2021-08-10 Gene therapies for lysosomal disorders

Publications (1)

Publication Number Publication Date
CN116157527A true CN116157527A (zh) 2023-05-23

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CN202180062323.1A Pending CN116157527A (zh) 2020-08-10 2021-08-10 用于溶酶体病症的基因疗法

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US (1) US20230310654A1 (https=)
EP (1) EP4192516A2 (https=)
JP (1) JP2023537903A (https=)
KR (1) KR20230051529A (https=)
CN (1) CN116157527A (https=)
AU (1) AU2021324686A1 (https=)
CA (1) CA3190866A1 (https=)
IL (1) IL300407A (https=)
MX (1) MX2023001562A (https=)
WO (1) WO2022035903A2 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018346104B2 (en) 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US20250205365A1 (en) * 2022-03-18 2025-06-26 University Of Florida Research Foundation, Incorporated Methods and compositions for treating rbm20 related cardiomyopathy with a viral vector
WO2024229161A1 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
US20250152747A1 (en) * 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating gaucher disease and gba-parkinson’s disease
WO2025229340A1 (en) 2024-05-01 2025-11-06 Spur Therapeutics Limited Methods and compositions for treatment of a synucleinopathy and/or a disease associated with a gcase deficiency
KR20260021150A (ko) 2024-08-05 2026-02-13 충북대학교 산학협력단 Gba1 돌연변이 보인자의 dj-1 발현수준에 따른 파킨슨병의 위험도 예측 방법

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008024998A2 (en) 2006-08-24 2008-02-28 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
EP3204503A1 (en) * 2014-10-06 2017-08-16 Arthrogen BV Aav-based gene therapy
HK1256341A1 (zh) * 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
JP7496667B2 (ja) 2016-04-21 2024-06-07 ビロベク，インコーポレイテッド 昆虫細胞中でのａａｖ生成、方法およびその組成物
CA3068328A1 (en) * 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
AU2018346104B2 (en) * 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112020006661A2 (pt) * 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
JP7664848B2 (ja) * 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法

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WO2022035903A3 (en) 2022-09-29
US20230310654A1 (en) 2023-10-05
IL300407A (en) 2023-04-01
AU2021324686A1 (en) 2023-04-13
WO2022035903A2 (en) 2022-02-17
KR20230051529A (ko) 2023-04-18
JP2023537903A (ja) 2023-09-06
MX2023001562A (es) 2023-05-03
EP4192516A2 (en) 2023-06-14
CA3190866A1 (en) 2022-02-17

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