JP2023534293A5 - - Google Patents

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Publication number
JP2023534293A5
JP2023534293A5 JP2023503074A JP2023503074A JP2023534293A5 JP 2023534293 A5 JP2023534293 A5 JP 2023534293A5 JP 2023503074 A JP2023503074 A JP 2023503074A JP 2023503074 A JP2023503074 A JP 2023503074A JP 2023534293 A5 JP2023534293 A5 JP 2023534293A5
Authority
JP
Japan
Prior art keywords
human
aav
fmrp
vector
promoter
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023503074A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023534293A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/041975 external-priority patent/WO2022016055A1/en
Publication of JP2023534293A publication Critical patent/JP2023534293A/ja
Publication of JP2023534293A5 publication Critical patent/JP2023534293A5/ja
Pending legal-status Critical Current

Links

JP2023503074A 2020-07-17 2021-07-16 脆弱x症候群の治療のための方法及び組成物 Pending JP2023534293A (ja)

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

Publications (2)

Publication Number Publication Date
JP2023534293A JP2023534293A (ja) 2023-08-08
JP2023534293A5 true JP2023534293A5 (enExample) 2024-07-24

Family

ID=79554278

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023503074A Pending JP2023534293A (ja) 2020-07-17 2021-07-16 脆弱x症候群の治療のための方法及び組成物

Country Status (9)

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

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4497436A4 (en) * 2022-03-22 2025-08-06 Univ Kyoto 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
EP4731267A1 (en) 2023-06-23 2026-04-29 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
EP4491733A3 (en) * 2015-04-23 2025-03-26 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
EP3856910B1 (en) * 2018-09-24 2024-11-20 Merck Sharp & Dohme LLC Expression vectors for eukaryotic expression systems

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