JP2021514956A - コハク酸セミアルデヒドデヒドロゲナーゼ欠損症（ｓｓａｄｈｄ）を処置するための組成物および方法 - Google Patents

コハク酸セミアルデヒドデヒドロゲナーゼ欠損症（ｓｓａｄｈｄ）を処置するための組成物および方法 Download PDF

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JP2021514956A
JP2021514956A JP2020544456A JP2020544456A JP2021514956A JP 2021514956 A JP2021514956 A JP 2021514956A JP 2020544456 A JP2020544456 A JP 2020544456A JP 2020544456 A JP2020544456 A JP 2020544456A JP 2021514956 A JP2021514956 A JP 2021514956A
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gaba
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composition
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ssadh
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カーラ レイン ヴォーゲル
カーラ レイン ヴォーゲル
ケネス マイケル ギブソン
ケネス マイケル ギブソン
ギャレット ロバート エインズリー
ギャレット ロバート エインズリー
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ワシントン ステイト ユニバーシティー
ワシントン ステイト ユニバーシティー
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    • C12Y102/01024—Succinate-semialdehyde dehydrogenase (NAD+) (1.2.1.24)

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JP2020544456A 2018-02-22 2019-02-12 コハク酸セミアルデヒドデヒドロゲナーゼ欠損症（ｓｓａｄｈｄ）を処置するための組成物および方法 Pending JP2021514956A (ja)

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JP2023139463A JP2023162344A (ja) 2018-02-22 2023-08-30 コハク酸セミアルデヒドデヒドロゲナーゼ欠損症（ｓｓａｄｈｄ）を処置するための組成物および方法

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US201862634092P 2018-02-22 2018-02-22
US62/634,092 2018-02-22
PCT/US2019/017623 WO2019164703A1 (en) 2018-02-22 2019-02-12 Compositions and methods for treating succinic semialdehyde dehydrogenase deficiency (ssadhd)

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JP2023139463A Pending JP2023162344A (ja) 2018-02-22 2023-08-30 コハク酸セミアルデヒドデヒドロゲナーゼ欠損症（ｓｓａｄｈｄ）を処置するための組成物および方法

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KR (1) KR20200140255A (https=)
AU (1) AU2019225729A1 (https=)
CA (1) CA3092035A1 (https=)
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Publication number Priority date Publication date Assignee Title
US20250018061A1 (en) * 2021-12-02 2025-01-16 The Children`S Medical Center Corporation Gene therapy in succinic semialdehyde dehydrogenase deficiency (ssadhd)
CN116478961B (zh) * 2023-04-27 2023-09-15 北京因诺惟康医药科技有限公司 CRISPR/SprCas9基因编辑系统的开发及应用
WO2025042900A1 (en) * 2023-08-21 2025-02-27 Washington State University Messenger rna treatment for succinic semialdehyde dehydrogenase deficiency

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
BIOCHIM BIOPHYS ACTA, vol. 1863, no. 1, JPN6023007903, January 2017 (2017-01-01), pages 33 - 42, ISSN: 0005205821 *
MOLECULAR THERAPY, vol. 9, no. 4, JPN6023007904, 2004, pages 527 - 539, ISSN: 0005205820 *

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US20220331448A1 (en) 2022-10-20
CA3092035A1 (en) 2019-08-29
US20210008225A1 (en) 2021-01-14
WO2019164703A1 (en) 2019-08-29
JP2023162344A (ja) 2023-11-08
KR20200140255A (ko) 2020-12-15

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