CN114072514A - 用于治疗atp酶介导的疾病的组合物和方法 - Google Patents

用于治疗atp酶介导的疾病的组合物和方法 Download PDF

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CN114072514A
CN114072514A CN202080047216.7A CN202080047216A CN114072514A CN 114072514 A CN114072514 A CN 114072514A CN 202080047216 A CN202080047216 A CN 202080047216A CN 114072514 A CN114072514 A CN 114072514A
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vector
nucleic acid
promoter
seq
group
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M·米卡蒂
A·胡南扬
B·坎托尔
A·阿索坎
R·普拉纳姆
D·科贝尔勒
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Duke University
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12Y306/01—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
    • C12Y306/0103—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1) m7G(5')pppN diphosphatase (3.6.1.30)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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CN202080047216.7A 2019-05-14 2020-05-14 用于治疗atp酶介导的疾病的组合物和方法 Pending CN114072514A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962847416P 2019-05-14 2019-05-14
US62/847,416 2019-05-14
PCT/US2020/032978 WO2020232297A1 (en) 2019-05-14 2020-05-14 Compositions and methods for the treatment of atpase-mediated diseases

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CN114072514A true CN114072514A (zh) 2022-02-18

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US (2) US11717560B2 (https=)
EP (1) EP3969597A4 (https=)
JP (1) JP2022531809A (https=)
KR (1) KR20220032005A (https=)
CN (1) CN114072514A (https=)
AU (1) AU2020274200A1 (https=)
BR (1) BR112021022951A2 (https=)
CA (1) CA3140049A1 (https=)
IL (1) IL287796A (https=)
MX (1) MX2021013913A (https=)
SG (1) SG11202112078TA (https=)
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AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
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US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
TW202323528A (zh) * 2021-10-18 2023-06-16 美商邏輯生物療法公司 用於治療威爾森氏病之基因療法
US20250320509A1 (en) * 2022-05-23 2025-10-16 The Johns Hopkins University Compositions and methods for treating wilson's disease

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Publication number Priority date Publication date Assignee Title
CN115851730A (zh) * 2022-11-16 2023-03-28 泰达国际心血管病医院 Myh6基因启动子变异及其应用与先天性心脏病基因检测试剂盒

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US20240075111A1 (en) 2024-03-07
AU2020274200A1 (en) 2021-11-25
US20220088152A1 (en) 2022-03-24
SG11202112078TA (en) 2021-11-29
EP3969597A4 (en) 2023-01-25
IL287796A (en) 2022-01-01
KR20220032005A (ko) 2022-03-15
JP2022531809A (ja) 2022-07-11
EP3969597A1 (en) 2022-03-23
US12533397B2 (en) 2026-01-27
US11717560B2 (en) 2023-08-08
MX2021013913A (es) 2022-03-17
CA3140049A1 (en) 2020-11-19
WO2020232297A1 (en) 2020-11-19

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