JP2023524247A - 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 - Google Patents

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 Download PDF

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JP2023524247A
JP2023524247A JP2022566154A JP2022566154A JP2023524247A JP 2023524247 A JP2023524247 A JP 2023524247A JP 2022566154 A JP2022566154 A JP 2022566154A JP 2022566154 A JP2022566154 A JP 2022566154A JP 2023524247 A JP2023524247 A JP 2023524247A
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ube3a
expression
vector
ats
aav
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JP2023524247A5 (enExample
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ウイルソン，ジェームス・エム
シュミッド，ラルフ
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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
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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)

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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

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

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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

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017075335A1 (en) * 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
JP2019503171A (ja) * 2015-12-24 2019-02-07 オキシレイン ユーケー リミテッド ヒトα−Ｎ−アセチルガラクトサミニダーゼポリペプチド
WO2019168950A1 (en) * 2018-02-27 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
WO2020068990A1 (en) * 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy

Family Cites Families (4)

* 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
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

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017075335A1 (en) * 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
JP2019503171A (ja) * 2015-12-24 2019-02-07 オキシレイン ユーケー リミテッド ヒトα−Ｎ−アセチルガラクトサミニダーゼポリペプチド
WO2019168950A1 (en) * 2018-02-27 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
WO2020068990A1 (en) * 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MENG LINYAN ET AL.: "Truncation of Ube3a-ATS Unsilences Paternal Ube3a and Ameliorates Behavioral Defects in the Angelman", PLOS GENETICS, vol. Vol.9, Issue 12, e1004039, JPN6025054159, 26 December 2013 (2013-12-26), pages 1 - 13, ISSN: 0005893767 *

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US20230167438A1 (en) 2023-06-01
EP4142802A4 (en) 2024-07-24
EP4142802A1 (en) 2023-03-08

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