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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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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JP2022501044A JP2022501044A (ja) 2022-01-06
JP2022501044A5 JP2022501044A5 (https=) 2022-09-28
JPWO2020061528A5 JPWO2020061528A5 (https=) 2022-09-28
JP7546295B2 true JP7546295B2 (ja) 2024-09-06

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CA3113648A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
EP4142802A4 (en) * 2020-04-28 2024-07-24 The Trustees of The University of Pennsylvania COMPOSITIONS AND USES THEREOF FOR THE 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
IL315359A (en) * 2022-03-07 2024-11-01 Univ Connecticut shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
KR20230152608A (ko) 2022-04-27 2023-11-03 중앙대학교 산학협력단 CRISPR-Cas12f1 시스템을 이용한 유전체의 단일 뉴클레오타이드 편집 방법 및 이의 용도

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