JP7546295B2 - 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 - Google Patents

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 Download PDF

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JP7546295B2
JP7546295B2 JP2021515639A JP2021515639A JP7546295B2 JP 7546295 B2 JP7546295 B2 JP 7546295B2 JP 2021515639 A JP2021515639 A JP 2021515639A JP 2021515639 A JP2021515639 A JP 2021515639A JP 7546295 B2 JP7546295 B2 JP 7546295B2
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particle
nucleotide sequence
shrna
polynucleotide
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JP2022501044A (ja
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ストーミー・チェンバレン
ノエル・ジャーメイン
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University of Connecticut
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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)

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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

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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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