AU2019344006B2 - Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome - Google Patents

Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome

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AU2019344006B2
AU2019344006B2 AU2019344006A AU2019344006A AU2019344006B2 AU 2019344006 B2 AU2019344006 B2 AU 2019344006B2 AU 2019344006 A AU2019344006 A AU 2019344006A AU 2019344006 A AU2019344006 A AU 2019344006A AU 2019344006 B2 AU2019344006 B2 AU 2019344006B2
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ube3a
polynucleotide
seq
shrna
rna
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AU2019344006A1 (en
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Stormy CHAMBERLAIN
Noelle GERMAIN
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University of Connecticut
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University of Connecticut
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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AU2019344006A 2018-09-21 2019-09-20 Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome Active AU2019344006B2 (en)

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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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CN (2) CN119592561A (enExample)
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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
JP2023524247A (ja) * 2020-04-28 2023-06-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アンジェルマン症候群の治療のための組成物及びその使用
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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JP7546295B2 (ja) 2024-09-06
CN113164624B (zh) 2024-09-24
EP3833398A4 (en) 2021-12-22
WO2020061528A1 (en) 2020-03-26
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US20210332368A1 (en) 2021-10-28
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