CN117136077A - 用于治疗脆性x综合征的方法和组合物 - Google Patents

用于治疗脆性x综合征的方法和组合物 Download PDF

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Publication number
CN117136077A
CN117136077A CN202180059850.7A CN202180059850A CN117136077A CN 117136077 A CN117136077 A CN 117136077A CN 202180059850 A CN202180059850 A CN 202180059850A CN 117136077 A CN117136077 A CN 117136077A
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aav
human
fmrp
vector
promoter
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CN202180059850.7A
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Chinese (zh)
Inventor
E·佩达帕蒂
C·格罗斯
C·埃里克森
D·迪斯穆克
E·K·德希尔瓦
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Fuge Biological Co
Cincinnati Childrens Hospital Medical Center
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Fuge Biological Co
Cincinnati Childrens Hospital Medical Center
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Publication of CN117136077A publication Critical patent/CN117136077A/zh
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CN202180059850.7A 2020-07-17 2021-07-16 用于治疗脆性x综合征的方法和组合物 Pending CN117136077A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063053461P 2020-07-17 2020-07-17
US63/053,461 2020-07-17
PCT/US2021/041975 WO2022016055A1 (en) 2020-07-17 2021-07-16 Methods and compositions for treatment of fragile x syndrome

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Publication Number Publication Date
CN117136077A true CN117136077A (zh) 2023-11-28

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CN202180059850.7A Pending CN117136077A (zh) 2020-07-17 2021-07-16 用于治疗脆性x综合征的方法和组合物

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US (1) US20230295654A1 (https=)
EP (1) EP4181877A4 (https=)
JP (1) JP2023534293A (https=)
KR (1) KR20230084465A (https=)
CN (1) CN117136077A (https=)
AU (1) AU2021308666A1 (https=)
CA (1) CA3189657A1 (https=)
IL (1) IL299889A (https=)
WO (1) WO2022016055A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250195497A1 (en) * 2022-03-22 2025-06-19 Kyoto University Therapeutic or prophylactic medicine for fragile x syndrome
GB202206336D0 (en) * 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
AU2024313758A1 (en) 2023-06-23 2026-01-08 Uniqure Biopharma B.V. Novel fragile x constructs

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012056440A1 (en) * 2010-10-28 2012-05-03 Nanodoc Ltd. COMPOSITIONS AND METHODS FOR ACTIVATING EXPRESSION BY A SPECIFIC ENDOGENOUS miRNA
CA3019315A1 (en) * 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
GB2574243A (en) * 2018-05-31 2019-12-04 The Govening Council Of The Univ Of Toronto Adeno-associated viral vector-mediated gene therapy for treating fragile X-associated disorders
US12421524B2 (en) * 2018-09-24 2025-09-23 Merck Sharp & Dohme Llc Expression vectors for eukaryotic expression systems

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Publication number Publication date
EP4181877A1 (en) 2023-05-24
JP2023534293A (ja) 2023-08-08
IL299889A (en) 2023-03-01
WO2022016055A1 (en) 2022-01-20
US20230295654A1 (en) 2023-09-21
CA3189657A1 (en) 2022-01-20
EP4181877A4 (en) 2024-10-23
KR20230084465A (ko) 2023-06-13
AU2021308666A1 (en) 2023-02-16

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