JP7769355B2 - ミトコンドリアｄｎａ枯渇症候群を含む不均衡ヌクレオチドプールに起因する疾患の遺伝子治療 - Google Patents

ミトコンドリアｄｎａ枯渇症候群を含む不均衡ヌクレオチドプールに起因する疾患の遺伝子治療

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JP7769355B2
JP7769355B2 JP2020557222A JP2020557222A JP7769355B2 JP 7769355 B2 JP7769355 B2 JP 7769355B2 JP 2020557222 A JP2020557222 A JP 2020557222A JP 2020557222 A JP2020557222 A JP 2020557222A JP 7769355 B2 JP7769355 B2 JP 7769355B2
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acid sequence
nucleic acid
amino acid
seq
day
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JP2021522173A (ja
JP2021522173A5 (enExample
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ヒラノ，ミチオ
アクマン，ハサン，オー．
ロペス－ゴメス，カルロス
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Columbia University in the City of New York
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    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
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    • A61K31/7072—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
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    • C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
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JP2020557222A 2018-04-18 2019-04-18 ミトコンドリアｄｎａ枯渇症候群を含む不均衡ヌクレオチドプールに起因する疾患の遺伝子治療 Active JP7769355B2 (ja)

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US201862659281P 2018-04-18 2018-04-18
US62/659,281 2018-04-18
PCT/US2019/028108 WO2019204593A1 (en) 2018-04-18 2019-04-18 Gene therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes

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JP2021522173A JP2021522173A (ja) 2021-08-30
JP2021522173A5 JP2021522173A5 (enExample) 2022-04-18
JP7769355B2 true JP7769355B2 (ja) 2025-11-13

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US (1) US12419970B2 (enExample)
EP (1) EP3781671A4 (enExample)
JP (1) JP7769355B2 (enExample)
KR (1) KR20210009317A (enExample)
CN (1) CN112312929A (enExample)
AU (1) AU2019255745A1 (enExample)
BR (1) BR112020021324A2 (enExample)
CA (1) CA3097392A1 (enExample)
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EA202191079A1 (ru) 2018-11-19 2021-09-20 Зодженикс Интернэшнл Лимитед Способы лечения синдрома ретта с применением фенфлурамина
US20220403417A1 (en) * 2019-11-20 2022-12-22 University Of Massachusetts Aav-based delivery of thymine kinase 2
US11612574B2 (en) 2020-07-17 2023-03-28 Zogenix International Limited Method of treating patients infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)
US20220096514A1 (en) * 2020-09-29 2022-03-31 Zogenix International Limited Methods of treating thymidine kinase 2 deficiency by administering deoxycytidine and deoxythymidine
WO2022204555A1 (en) * 2021-03-26 2022-09-29 Zogenix Mds, Inc. Aqueous solutions containing purines and pyrimidines and uses thereof
CN121443327A (zh) * 2023-06-30 2026-01-30 社会福祉法人 三星生命公益财团 包含搭载水螅NDi1的重组腺相关病毒载体的用于预防、改善或治疗线粒体复合物1异常疾病的组合物
CN120060299B (zh) * 2025-02-21 2025-11-28 三峡大学 一种密码子优化的twnk基因、腺相关病毒载体及其应用

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IL278130A (en) 2020-12-31
US20210100917A1 (en) 2021-04-08
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