JPWO2021221995A5 - - Google Patents

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Publication number
JPWO2021221995A5
JPWO2021221995A5 JP2022566010A JP2022566010A JPWO2021221995A5 JP WO2021221995 A5 JPWO2021221995 A5 JP WO2021221995A5 JP 2022566010 A JP2022566010 A JP 2022566010A JP 2022566010 A JP2022566010 A JP 2022566010A JP WO2021221995 A5 JPWO2021221995 A5 JP WO2021221995A5
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JP
Japan
Prior art keywords
seq
nucleic acid
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
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English (en)
Japanese (ja)
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JP7821742B2 (ja
JP2023524024A (ja
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 JPWO2021221995A5 publication Critical patent/JPWO2021221995A5/ja
Application granted granted Critical
Publication of JP7821742B2 publication Critical patent/JP7821742B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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
JPWO2021221995A5 true JPWO2021221995A5 (enExample) 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 (enExample)
EP (1) EP4142759A4 (enExample)
JP (1) JP7821742B2 (enExample)
KR (1) KR20230003012A (enExample)
CN (1) CN115461066B (enExample)
AU (1) AU2021263534A1 (enExample)
BR (1) BR112022021964A2 (enExample)
CA (1) CA3174070A1 (enExample)
IL (1) IL297605A (enExample)
TW (1) TW202206599A (enExample)
WO (1) WO2021221995A1 (enExample)

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
WO2023221942A1 (en) 2022-05-16 2023-11-23 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating glutaric aciduria type i
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
WO2002059381A2 (en) 2001-01-06 2002-08-01 The General Hospital Corporation Gene for identifying individuals with familial dysautonomia
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
EP2900686B1 (en) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
WO2017070525A1 (en) * 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
US11078247B2 (en) * 2016-05-04 2021-08-03 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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