JP2022501044A5 - - Google Patents

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Publication number
JP2022501044A5
JP2022501044A5 JP2021515639A JP2021515639A JP2022501044A5 JP 2022501044 A5 JP2022501044 A5 JP 2022501044A5 JP 2021515639 A JP2021515639 A JP 2021515639A JP 2021515639 A JP2021515639 A JP 2021515639A JP 2022501044 A5 JP2022501044 A5 JP 2022501044A5
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JP
Japan
Prior art keywords
polynucleotide
particle
seq
nucleotide sequence
ribozyme
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JP2021515639A
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English (en)
Japanese (ja)
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JP2022501044A (ja
JP7546295B2 (ja
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Priority claimed from PCT/US2019/052272 external-priority patent/WO2020061528A1/en
Publication of JP2022501044A publication Critical patent/JP2022501044A/ja
Publication of JP2022501044A5 publication Critical patent/JP2022501044A5/ja
Application granted granted Critical
Publication of JP7546295B2 publication Critical patent/JP7546295B2/ja
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JP2021515639A 2018-09-21 2019-09-20 ヒトアンジェルマン症候群において父方ube3a遺伝子発現を復活させるための組成物および方法 Active JP7546295B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862734435P 2018-09-21 2018-09-21
US62/734,435 2018-09-21
PCT/US2019/052272 WO2020061528A1 (en) 2018-09-21 2019-09-20 Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome

Publications (3)

Publication Number Publication Date
JP2022501044A JP2022501044A (ja) 2022-01-06
JP2022501044A5 true JP2022501044A5 (enExample) 2022-09-28
JP7546295B2 JP7546295B2 (ja) 2024-09-06

Family

ID=69887937

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021515639A Active JP7546295B2 (ja) 2018-09-21 2019-09-20 ヒトアンジェルマン症候群において父方ube3a遺伝子発現を復活させるための組成物および方法

Country Status (10)

Country Link
US (2) US12221609B2 (enExample)
EP (1) EP3833398A4 (enExample)
JP (1) JP7546295B2 (enExample)
KR (1) KR102782761B1 (enExample)
CN (2) CN119592561A (enExample)
AU (2) AU2019344006B2 (enExample)
CA (1) CA3113648A1 (enExample)
IL (1) IL281563B2 (enExample)
MX (1) MX2021003303A (enExample)
WO (1) WO2020061528A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3833398A4 (en) 2018-09-21 2021-12-22 University of Connecticut COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS
WO2021222232A1 (en) * 2020-04-28 2021-11-04 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
US20240318198A1 (en) * 2021-02-03 2024-09-26 The Board Of Regents Of The University Of Texas System Gene therapy for angelman syndrome
CN119384504A (zh) * 2022-03-07 2025-01-28 康涅狄格大学 靶向SNORD115位置以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA
KR102986878B1 (ko) 2022-04-27 2026-07-06 중앙대학교 산학협력단 CRISPR-Cas12f1 시스템을 이용한 유전체의 단일 뉴클레오타이드 편집 방법 및 이의 용도

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