JP2023524247A5 - - Google Patents

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Publication number
JP2023524247A5
JP2023524247A5 JP2022566154A JP2022566154A JP2023524247A5 JP 2023524247 A5 JP2023524247 A5 JP 2023524247A5 JP 2022566154 A JP2022566154 A JP 2022566154A JP 2022566154 A JP2022566154 A JP 2022566154A JP 2023524247 A5 JP2023524247 A5 JP 2023524247A5
Authority
JP
Japan
Prior art keywords
ube3a
expression cassette
raav
sequence
ats
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022566154A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023524247A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/029378 external-priority patent/WO2021222232A1/en
Publication of JP2023524247A publication Critical patent/JP2023524247A/ja
Publication of JP2023524247A5 publication Critical patent/JP2023524247A5/ja
Pending legal-status Critical Current

Links

JP2022566154A 2020-04-28 2021-04-27 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 Pending JP2023524247A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063016712P 2020-04-28 2020-04-28
US63/016,712 2020-04-28
US202063118299P 2020-11-25 2020-11-25
US63/118,299 2020-11-25
PCT/US2021/029378 WO2021222232A1 (en) 2020-04-28 2021-04-27 Compositions and uses thereof for treatment of angelman syndrome

Publications (2)

Publication Number Publication Date
JP2023524247A JP2023524247A (ja) 2023-06-09
JP2023524247A5 true JP2023524247A5 (enExample) 2024-05-09

Family

ID=78332167

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022566154A Pending JP2023524247A (ja) 2020-04-28 2021-04-27 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

Country Status (4)

Country Link
US (1) US20230167438A1 (enExample)
EP (1) EP4142802A4 (enExample)
JP (1) JP2023524247A (enExample)
WO (1) WO2021222232A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023184108A1 (en) * 2022-03-28 2023-10-05 Huigene Therapeutics Co., Ltd. Crispr-cas13 system for treating ube3a-associated diseases

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2809199T3 (es) * 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
JP6851319B2 (ja) * 2015-04-27 2021-03-31 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 ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系
CA2984629C (en) * 2015-05-07 2024-06-18 University Of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
US20180230489A1 (en) * 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
KR20180088919A (ko) * 2015-12-24 2018-08-07 옥시레인 유케이 리미티드 인간 알파-n-아세틸갈락토사미니다아제 폴리펩티드
EP3758714A4 (en) * 2018-02-27 2021-12-01 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR TREATMENT OF ANGELMAN'S SYNDROME
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
US11149256B2 (en) * 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy

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