TW202302855A - 用於治療法布瑞氏症之組成物及方法 - Google Patents

用於治療法布瑞氏症之組成物及方法 Download PDF

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TW202302855A
TW202302855A TW111107143A TW111107143A TW202302855A TW 202302855 A TW202302855 A TW 202302855A TW 111107143 A TW111107143 A TW 111107143A TW 111107143 A TW111107143 A TW 111107143A TW 202302855 A TW202302855 A TW 202302855A
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vector
itr
raav
seq
promoter
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TW111107143A
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Chinese (zh)
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里茲瓦納 伊斯蘭
穆哈 拉賓卓 迪喜潘德
瑪徐蘇丹 納塔拉珍
隆熙 朴
薇薇安 趙
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日商武田藥品工業股份有限公司
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Publication of TW202302855A publication Critical patent/TW202302855A/zh

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    • 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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    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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TW111107143A 2021-02-26 2022-02-25 用於治療法布瑞氏症之組成物及方法 TW202302855A (zh)

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US202163154485P 2021-02-26 2021-02-26
US63/154,485 2021-02-26

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TW202302855A true TW202302855A (zh) 2023-01-16

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US (1) US20240175054A1 (https=)
EP (1) EP4298227A1 (https=)
JP (1) JP2024509792A (https=)
KR (1) KR20230150836A (https=)
CN (1) CN117321212A (https=)
AR (1) AR124981A1 (https=)
AU (1) AU2022227017A1 (https=)
BR (1) BR112023016983A2 (https=)
CA (1) CA3209673A1 (https=)
CL (1) CL2023002530A1 (https=)
CO (1) CO2023011012A2 (https=)
EC (1) ECSP23072174A (https=)
IL (1) IL305440A (https=)
MX (1) MX2023009978A (https=)
PE (1) PE20240806A1 (https=)
TW (1) TW202302855A (https=)
WO (1) WO2022183052A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025529884A (ja) * 2022-08-25 2025-09-09 武田薬品工業株式会社 ファブリー病の治療に使用するための組成物
CN121002183A (zh) * 2023-04-17 2025-11-21 杭州复因生物科技有限公司 用于基因治疗的组合物和方法
WO2024236544A1 (en) 2023-05-17 2024-11-21 Takeda Pharmaceutical Company Limited Pet tracer compounds for tracing alpha-galactosidase a and reaction intermediates, and methods of use

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US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
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US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
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WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
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DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
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CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
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AU2021356684A1 (en) * 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease

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CO2023011012A2 (es) 2023-08-28
PE20240806A1 (es) 2024-04-18
MX2023009978A (es) 2023-09-06
US20240175054A1 (en) 2024-05-30
AU2022227017A1 (en) 2023-09-07
ECSP23072174A (es) 2023-10-31
AR124981A1 (es) 2023-05-24
CL2023002530A1 (es) 2024-04-19
KR20230150836A (ko) 2023-10-31
IL305440A (en) 2023-10-01
EP4298227A1 (en) 2024-01-03
CN117321212A (zh) 2023-12-29
JP2024509792A (ja) 2024-03-05
CA3209673A1 (en) 2022-09-01
BR112023016983A2 (pt) 2023-11-07
WO2022183052A1 (en) 2022-09-01

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