KR20220140537A - Cdkl5 결핍 장애를 치료하기 위한 유전자 요법 - Google Patents

Cdkl5 결핍 장애를 치료하기 위한 유전자 요법 Download PDF

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KR20220140537A
KR20220140537A KR1020227029963A KR20227029963A KR20220140537A KR 20220140537 A KR20220140537 A KR 20220140537A KR 1020227029963 A KR1020227029963 A KR 1020227029963A KR 20227029963 A KR20227029963 A KR 20227029963A KR 20220140537 A KR20220140537 A KR 20220140537A
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raav
sequence
promoter
ser
cdkl5
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샤릴 린 파이피-마리시흐
매튜 스콧 풀러
마가렛 캐롤라인 라이트
로렐라이 이오아나 스토이카
스튜어트 크레이그
션 크리스토퍼 도허티
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울트라제닉스 파마수티컬 인코포레이티드
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11022—Cyclin-dependent kinase (2.7.11.22)

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020227029963A 2020-02-14 2021-02-11 Cdkl5 결핍 장애를 치료하기 위한 유전자 요법 Pending KR20220140537A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062976483P 2020-02-14 2020-02-14
US62/976,483 2020-02-14
US202063090492P 2020-10-12 2020-10-12
US63/090,492 2020-10-12
PCT/US2021/017656 WO2021163322A1 (en) 2020-02-14 2021-02-11 Gene therapy for treating cdkl5 deficiency disorder

Publications (1)

Publication Number Publication Date
KR20220140537A true KR20220140537A (ko) 2022-10-18

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KR1020227029963A Pending KR20220140537A (ko) 2020-02-14 2021-02-11 Cdkl5 결핍 장애를 치료하기 위한 유전자 요법

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US (1) US20230054144A1 (https=)
EP (1) EP4103724A1 (https=)
JP (1) JP2023514204A (https=)
KR (1) KR20220140537A (https=)
CN (1) CN115151648A (https=)
AU (1) AU2021218703A1 (https=)
BR (1) BR112022015921A2 (https=)
CA (1) CA3164714A1 (https=)
IL (1) IL294781A (https=)
MX (1) MX2022009982A (https=)
TW (1) TWI904135B (https=)
WO (1) WO2021163322A1 (https=)

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WO2022225978A1 (en) * 2021-04-21 2022-10-27 The Regents Of The University Of California Use of a split dcas fusion protein system for epigenetic editing
EP4387641A4 (en) * 2021-08-18 2025-07-09 Ultragenyx Pharmaceutical Inc RECOMBINANT AAV FORMULATIONS
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CN116377050A (zh) * 2022-12-16 2023-07-04 湖南家辉生物技术有限公司 一种发育性癫痫性脑病2型致病基因cdkl5突变位点的应用及其检测试剂和应用
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TWI904135B (zh) 2025-11-11
WO2021163322A1 (en) 2021-08-19
CN115151648A (zh) 2022-10-04
IL294781A (en) 2022-09-01
EP4103724A1 (en) 2022-12-21
TW202144577A (zh) 2021-12-01
JP2023514204A (ja) 2023-04-05
US20230054144A1 (en) 2023-02-23
AU2021218703A1 (en) 2022-07-28
BR112022015921A2 (pt) 2022-10-04
CA3164714A1 (en) 2021-08-19
MX2022009982A (es) 2022-09-12

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