CN119592561A - 恢复人类快乐木偶综合征中父系ube3a基因表达的组合物和方法 - Google Patents

恢复人类快乐木偶综合征中父系ube3a基因表达的组合物和方法 Download PDF

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CN119592561A
CN119592561A CN202411375936.2A CN202411375936A CN119592561A CN 119592561 A CN119592561 A CN 119592561A CN 202411375936 A CN202411375936 A CN 202411375936A CN 119592561 A CN119592561 A CN 119592561A
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seq
ribozyme
polynucleotide
ube3a
particle
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斯托米·张伯伦
诺艾儿·杰曼
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University of Connecticut
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University of Connecticut
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CN202411375936.2A 2018-09-21 2019-09-20 恢复人类快乐木偶综合征中父系ube3a基因表达的组合物和方法 Pending CN119592561A (zh)

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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
CN201980076741.9A CN113164624B (zh) 2018-09-21 2019-09-20 恢复人类快乐木偶综合征中父系ube3a基因表达的组合物和方法

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CN201980076741.9A Active CN113164624B (zh) 2018-09-21 2019-09-20 恢复人类快乐木偶综合征中父系ube3a基因表达的组合物和方法

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WO2020061528A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
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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
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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MX2021003303A (es) 2021-06-23
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US20210332368A1 (en) 2021-10-28
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