CN112312929A - 用于包括线粒体dna耗竭综合征在内的由不平衡的核苷酸库引起的疾病的基因疗法 - Google Patents

用于包括线粒体dna耗竭综合征在内的由不平衡的核苷酸库引起的疾病的基因疗法 Download PDF

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CN112312929A
CN112312929A CN201980039834.4A CN201980039834A CN112312929A CN 112312929 A CN112312929 A CN 112312929A CN 201980039834 A CN201980039834 A CN 201980039834A CN 112312929 A CN112312929 A CN 112312929A
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acid sequence
seq
nucleic acid
amino acid
transgene
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M·希拉诺
H·O·阿克曼
C·洛佩兹-戈麦斯
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Columbia University in the City of New York
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Columbia University in the City of New York
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CN201980039834.4A 2018-04-18 2019-04-18 用于包括线粒体dna耗竭综合征在内的由不平衡的核苷酸库引起的疾病的基因疗法 Pending CN112312929A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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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CN112312929A true CN112312929A (zh) 2021-02-02

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US (1) US12419970B2 (https=)
EP (1) EP3781671A4 (https=)
JP (1) JP7769355B2 (https=)
KR (1) KR20210009317A (https=)
CN (1) CN112312929A (https=)
AU (1) AU2019255745A1 (https=)
BR (1) BR112020021324A2 (https=)
CA (1) CA3097392A1 (https=)
CL (1) CL2020002704A1 (https=)
CO (1) CO2020014193A2 (https=)
IL (1) IL278130B1 (https=)
MX (1) MX2020010994A (https=)
WO (1) WO2019204593A1 (https=)

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WO2017112701A1 (en) 2015-12-22 2017-06-29 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
AU2017315273B2 (en) 2016-08-24 2021-04-29 Zogenix International Limited Formulation for inhibiting formation of 5-HT 2B agonists and methods of using same
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
EP3790537A1 (en) 2018-05-11 2021-03-17 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
AU2019384963B2 (en) 2018-11-19 2022-05-26 Zogenix International Limited Methods of treating Rett syndrome using fenfluramine
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
EP4313068A4 (en) 2021-03-26 2024-12-25 UCB Biosciences, Inc. Aqueous solutions containing purines and pyrimidines and uses thereof
KR20250004463A (ko) * 2023-06-30 2025-01-08 사회복지법인 삼성생명공익재단 Hydra NDi1가 탑재된 재조합 아데노부속바이러스 벡터를 포함하는, 미토콘드리아 복합체 1 이상질환의 예방, 개선 또는 치료용 조성물
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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US20210100917A1 (en) 2021-04-08
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KR20210009317A (ko) 2021-01-26
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