CN111770758A - 用于治疗Angelman综合征的方法和组合物 - Google Patents

用于治疗Angelman综合征的方法和组合物 Download PDF

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Publication number
CN111770758A
CN111770758A CN201980015559.2A CN201980015559A CN111770758A CN 111770758 A CN111770758 A CN 111770758A CN 201980015559 A CN201980015559 A CN 201980015559A CN 111770758 A CN111770758 A CN 111770758A
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China
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ube3a
subject
ats
nucleic acid
crispr
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Chinese (zh)
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M·J·泽尔卡
J·M·沃尔特
G·弗拉戈拉
J·M·西蒙
H·毛
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University of North Carolina at Chapel Hill
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University of North Carolina at Chapel Hill
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    • 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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CN201980015559.2A 2018-02-27 2019-02-27 用于治疗Angelman综合征的方法和组合物 Pending CN111770758A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862635815P 2018-02-27 2018-02-27
US62/635815 2018-02-27
PCT/US2019/019789 WO2019168950A1 (en) 2018-02-27 2019-02-27 Methods and compositions for treating angelman syndrome

Publications (1)

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CN111770758A true CN111770758A (zh) 2020-10-13

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CN201980015559.2A Pending CN111770758A (zh) 2018-02-27 2019-02-27 用于治疗Angelman综合征的方法和组合物

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US (1) US20210054370A1 (https=)
EP (1) EP3758714A4 (https=)
JP (1) JP2021513866A (https=)
CN (1) CN111770758A (https=)
CA (1) CA3091912A1 (https=)
WO (1) WO2019168950A1 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116004788A (zh) * 2022-08-01 2023-04-25 湖南家辉生物技术有限公司 一种Angelman综合症致病基因UBE3A突变位点的应用及其诊断试剂
WO2023185861A1 (en) * 2022-03-28 2023-10-05 Huidagene Therapeutics Co., Ltd. Guide nucleic acid targeting ube3a-ats and uses thereof
CN119177283A (zh) * 2024-11-25 2024-12-24 嘉检(广州)生物工程技术有限公司 用于pws/as快速诊断的引物组、试剂盒及系统

Families Citing this family (7)

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KR102922694B1 (ko) 2018-04-19 2026-02-03 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 유전자 편집을 위한 조성물 및 방법
EP3980533A1 (en) 2019-06-07 2022-04-13 Scribe Therapeutics Inc. Engineered casx systems
US20230042176A1 (en) * 2019-12-18 2023-02-09 National University Of Singapore Method for treating angelman syndrome and related disorders
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
KR20230128289A (ko) 2020-12-03 2023-09-04 스크라이브 테라퓨틱스 인크. 조작된 클래스 2 유형 v crispr 시스템
WO2023168000A1 (en) * 2022-03-03 2023-09-07 The Regents Of The University Of California Compositions and methods for treatment of angelman syndrome
WO2026006542A2 (en) * 2024-06-26 2026-01-02 Yale University Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome

Citations (2)

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WO2014004572A2 (en) * 2012-06-25 2014-01-03 Isis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
WO2014093622A2 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications

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EP3183358B1 (en) * 2014-08-19 2020-10-07 President and Fellows of Harvard College Rna-guided systems for probing and mapping of nucleic acids
US10400243B2 (en) * 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
WO2017048466A1 (en) * 2015-09-15 2017-03-23 The Regents Of The University Of California Compositions and methods for delivering biotherapeutics
US20190127713A1 (en) * 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2019084140A1 (en) * 2017-10-24 2019-05-02 Sangamo Therapeutics, Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES
KR102723989B1 (ko) * 2017-12-01 2024-11-01 더 텍사스 에이 & 엠 유니버시티 시스템 엔젤만 증후군 안티센스 치료
EP3720508A4 (en) * 2017-12-08 2021-09-01 University of Connecticut COMPOSITIONS AND METHODS FOR TREATMENT OF GENOMIC FOOTPRINT DISORDERS

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014004572A2 (en) * 2012-06-25 2014-01-03 Isis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
WO2014093622A2 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
BARBARA J BAILUS 等: "The prospect of molecular therapy for Angelman syndrome and other monogenic neurologic disorders" *
JACK SHUO-HUNG HSIAO: "Understanding the Regulation of UBE3A-ATS and the Process of UBE3A Imprinting Using iPSC Modeling and CRISPR/Cas9 Editing" *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023185861A1 (en) * 2022-03-28 2023-10-05 Huidagene Therapeutics Co., Ltd. Guide nucleic acid targeting ube3a-ats and uses thereof
WO2023184108A1 (en) * 2022-03-28 2023-10-05 Huigene Therapeutics Co., Ltd. Crispr-cas13 system for treating ube3a-associated diseases
CN116004788A (zh) * 2022-08-01 2023-04-25 湖南家辉生物技术有限公司 一种Angelman综合症致病基因UBE3A突变位点的应用及其诊断试剂
CN116004788B (zh) * 2022-08-01 2024-05-31 湖南家辉生物技术有限公司 一种Angelman综合症致病基因UBE3A突变位点的应用及其诊断试剂
CN119177283A (zh) * 2024-11-25 2024-12-24 嘉检(广州)生物工程技术有限公司 用于pws/as快速诊断的引物组、试剂盒及系统

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EP3758714A1 (en) 2021-01-06
US20210054370A1 (en) 2021-02-25
EP3758714A4 (en) 2021-12-01
JP2021513866A (ja) 2021-06-03
CA3091912A1 (en) 2019-09-06
WO2019168950A1 (en) 2019-09-06

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