TW202206599A - 用於基因療法的編碼天冬胺酸醯化酶（ａｓｐａ）之經修飾核酸及載體 - Google Patents

用於基因療法的編碼天冬胺酸醯化酶（ａｓｐａ）之經修飾核酸及載體 Download PDF

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TW202206599A
TW202206599A TW110115439A TW110115439A TW202206599A TW 202206599 A TW202206599 A TW 202206599A TW 110115439 A TW110115439 A TW 110115439A TW 110115439 A TW110115439 A TW 110115439A TW 202206599 A TW202206599 A TW 202206599A
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nucleic acid
seq
vector
aspa
acid sequence
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TW110115439A
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Chinese (zh)
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帕歐拉 里昂
傑瑞米 法蘭西斯
巴賽爾 塔里克 阿薩夫
修 淺野
凱瑟琳 哈里斯
艾里森 Ｐ 伯格
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美商輝瑞大藥廠
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    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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TW110115439A 2020-04-28 2021-04-28 用於基因療法的編碼天冬胺酸醯化酶（ａｓｐａ）之經修飾核酸及載體 TW202206599A (zh)

Applications Claiming Priority (4)

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

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Publication Number Publication Date
TW202206599A true TW202206599A (zh) 2022-02-16

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TW110115439A TW202206599A (zh) 2020-04-28 2021-04-28 用於基因療法的編碼天冬胺酸醯化酶（ａｓｐａ）之經修飾核酸及載體

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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

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* 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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US20230165977A1 (en) 2023-06-01
JP7821742B2 (ja) 2026-02-27
CN115461066B (zh) 2026-01-06
BR112022021964A2 (pt) 2023-03-14
EP4142759A4 (en) 2024-05-29
WO2021221995A1 (en) 2021-11-04
KR20230003012A (ko) 2023-01-05
AU2021263534A1 (en) 2023-01-19
CA3174070A1 (en) 2021-11-04
JP2023524024A (ja) 2023-06-08
EP4142759A1 (en) 2023-03-08
CN115461066A (zh) 2022-12-09
IL297605A (en) 2022-12-01

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