JP2023524024A5 - - Google Patents

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Publication number
JP2023524024A5
JP2023524024A5 JP2022566010A JP2022566010A JP2023524024A5 JP 2023524024 A5 JP2023524024 A5 JP 2023524024A5 JP 2022566010 A JP2022566010 A JP 2022566010A JP 2022566010 A JP2022566010 A JP 2022566010A JP 2023524024 A5 JP2023524024 A5 JP 2023524024A5
Authority
JP
Japan
Prior art keywords
nucleic acid
seq
acid sequence
vector
capsid
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.)
Granted
Application number
JP2022566010A
Other languages
English (en)
Japanese (ja)
Other versions
JP7821742B2 (ja
JP2023524024A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/028658 external-priority patent/WO2021221995A1/en
Publication of JP2023524024A publication Critical patent/JP2023524024A/ja
Publication of JP2023524024A5 publication Critical patent/JP2023524024A5/ja
Application granted granted Critical
Publication of JP7821742B2 publication Critical patent/JP7821742B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022566010A 2020-04-28 2021-04-22 アスパルトアシラーゼ(aspa)をコードする修飾核酸及び遺伝子治療のためのベクター Active JP7821742B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063016507P 2020-04-28 2020-04-28
US63/016,507 2020-04-28
US202063077144P 2020-09-11 2020-09-11
US63/077,144 2020-09-11
PCT/US2021/028658 WO2021221995A1 (en) 2020-04-28 2021-04-22 Modified nucleic acids encoding aspartoacylase (aspa) and vector for gene therapy

Publications (3)

Publication Number Publication Date
JP2023524024A JP2023524024A (ja) 2023-06-08
JP2023524024A5 true JP2023524024A5 (https=) 2024-05-01
JP7821742B2 JP7821742B2 (ja) 2026-02-27

Family

ID=78373856

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022566010A Active JP7821742B2 (ja) 2020-04-28 2021-04-22 アスパルトアシラーゼ(aspa)をコードする修飾核酸及び遺伝子治療のためのベクター

Country Status (11)

Country Link
US (1) US20230165977A1 (https=)
EP (1) EP4142759A4 (https=)
JP (1) JP7821742B2 (https=)
KR (1) KR20230003012A (https=)
CN (1) CN115461066B (https=)
AU (1) AU2021263534A1 (https=)
BR (1) BR112022021964A2 (https=)
CA (1) CA3174070A1 (https=)
IL (1) IL297605A (https=)
TW (1) TW202206599A (https=)
WO (1) WO2021221995A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3234809A1 (en) 2021-10-20 2023-04-27 Steven Goldman Isolated glial progenitor cells for use in the competition treatment of age-related white matter loss
EP4426331A1 (en) 2021-11-02 2024-09-11 University of Rochester Tcf7l2 mediated remyelination in the brain
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025129157A1 (en) * 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL156752A0 (en) 2001-01-06 2004-02-08 Gen Hospital Corp Gene for identifying individuals with familial dysautonomia
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
EP2900686B1 (en) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
EP3364997B1 (en) * 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
US10610606B2 (en) * 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof

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