JP2023520402A - プロピオン酸血症を処置するための遺伝子治療 - Google Patents

プロピオン酸血症を処置するための遺伝子治療 Download PDF

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JP2023520402A
JP2023520402A JP2022559589A JP2022559589A JP2023520402A JP 2023520402 A JP2023520402 A JP 2023520402A JP 2022559589 A JP2022559589 A JP 2022559589A JP 2022559589 A JP2022559589 A JP 2022559589A JP 2023520402 A JP2023520402 A JP 2023520402A
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マシュー スコット フラー，
サミュエル ウォズワース，
ケリー リード クラーク，
ショーン クリストファー ドーハティ，
スチュワート クレイグ，
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ウルトラジェニックス ファーマシューティカル インコーポレイテッド
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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JP2022559589A 2020-03-31 2021-03-30 プロピオン酸血症を処置するための遺伝子治療 Pending JP2023520402A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063002541P 2020-03-31 2020-03-31
US63/002,541 2020-03-31
PCT/US2021/024892 WO2021202532A1 (en) 2020-03-31 2021-03-30 Gene therapy for treating propionic acidemia

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JP2023520402A true JP2023520402A (ja) 2023-05-17
JP2023520402A5 JP2023520402A5 (https=) 2024-04-10

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US (1) US20230129893A1 (https=)
EP (1) EP4127189A1 (https=)
JP (1) JP2023520402A (https=)
AR (1) AR121709A1 (https=)
TW (1) TW202204624A (https=)
WO (1) WO2021202532A1 (https=)

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Publication number Priority date Publication date Assignee Title
BR112021006052A2 (pt) 2018-10-01 2021-09-08 Ultragenyx Pharmaceutical Inc. Terapia gênica para tratar acidemia propiônica
WO2022198079A1 (en) * 2021-03-19 2022-09-22 Mayo Foundation For Medical Education And Research Methods and materials for treating propionic acidemia
WO2023130003A2 (en) * 2021-12-29 2023-07-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Improved gene therapy constructs for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha

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JP2020506705A (ja) * 2017-02-01 2020-03-05 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア シトルリン血症を処置するための遺伝子療法

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US20230129893A1 (en) 2023-04-27
TW202204624A (zh) 2022-02-01
WO2021202532A1 (en) 2021-10-07
EP4127189A1 (en) 2023-02-08
AR121709A1 (es) 2022-06-29

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