JP2021517168A - パーキンソン病を処置するための組成物及び方法 - Google Patents

パーキンソン病を処置するための組成物及び方法 Download PDF

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JP2021517168A
JP2021517168A JP2020571336A JP2020571336A JP2021517168A JP 2021517168 A JP2021517168 A JP 2021517168A JP 2020571336 A JP2020571336 A JP 2020571336A JP 2020571336 A JP2020571336 A JP 2020571336A JP 2021517168 A JP2021517168 A JP 2021517168A
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acid sequence
gba
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amino acid
nucleic acid
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JPWO2019173756A5 (https=
JP2021517168A5 (https=
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メイソン，クリス
ティル，ニコ，ペーター ヴァン
ティル，ニコ，ペーター ヴァン
クーパー，オリバー
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アブロバイオ，インコーポレーテッド
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JP2020571336A 2018-03-09 2019-03-08 パーキンソン病を処置するための組成物及び方法 Pending JP2021517168A (ja)

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US201862641012P 2018-03-09 2018-03-09
US62/641,012 2018-03-09
PCT/US2019/021422 WO2019173756A1 (en) 2018-03-09 2019-03-08 Compositions and methods for treating parkinson's disease

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WO2021097131A1 (en) * 2019-11-12 2021-05-20 Orchard Therapeutics (Europe) Limited Compositions and methods for treating or preventing crohn's disease
KR20230003492A (ko) * 2020-03-29 2023-01-06 예다 리서치 앤드 디벨럽먼트 캄파니 리미티드 고셔병 치료용 베타-글루코세레브로시다아제 변이체
EP4146797A1 (en) * 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
WO2021239308A1 (en) * 2020-05-27 2021-12-02 Universität Zürich Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia
BR112023001456A2 (pt) * 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
WO2023004366A1 (en) * 2021-07-21 2023-01-26 Aspen Neuroscience, Inc. Transposon-based modulation of gba1 and related compositions and uses thereof
AU2024213347A1 (en) * 2023-02-02 2025-08-14 Embold Therapeutics, Inc. Engineered glucocerebrosidase variants
WO2024238448A2 (en) * 2023-05-12 2024-11-21 Temple University - Of The Commonwealth System Of Higher Education Chimeric capsid recombinant adeno-associated virus with bbb penetration and microglia targeting (aav-bm)
GB202307366D0 (en) * 2023-05-17 2023-06-28 Ospedale San Raffaele Srl Vector
EP4728055A2 (en) 2023-06-13 2026-04-22 Aspen Neuroscience, Inc. Methods for differentiating dopaminergic neurons from stem cells

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