JP2023551911A5 - - Google Patents

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Publication number
JP2023551911A5
JP2023551911A5 JP2023533671A JP2023533671A JP2023551911A5 JP 2023551911 A5 JP2023551911 A5 JP 2023551911A5 JP 2023533671 A JP2023533671 A JP 2023533671A JP 2023533671 A JP2023533671 A JP 2023533671A JP 2023551911 A5 JP2023551911 A5 JP 2023551911A5
Authority
JP
Japan
Prior art keywords
nucleic acid
ube3a
composition according
sequence
expression
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
JP2023533671A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023551911A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/061346 external-priority patent/WO2022119890A1/en
Publication of JP2023551911A publication Critical patent/JP2023551911A/ja
Publication of JP2023551911A5 publication Critical patent/JP2023551911A5/ja
Pending legal-status Critical Current

Links

JP2023533671A 2020-12-01 2021-12-01 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 JP2023551911A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063119860P 2020-12-01 2020-12-01
US63/119,860 2020-12-01
US202163179807P 2021-04-26 2021-04-26
US63/179,807 2021-04-26
PCT/US2021/061346 WO2022119890A1 (en) 2020-12-01 2021-12-01 Compositions and uses thereof for treatment of angelman syndrome

Publications (2)

Publication Number Publication Date
JP2023551911A JP2023551911A (ja) 2023-12-13
JP2023551911A5 true JP2023551911A5 (https=) 2024-12-09

Family

ID=80123326

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023533671A Pending JP2023551911A (ja) 2020-12-01 2021-12-01 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 (9)

Country Link
US (1) US20230414785A1 (https=)
EP (1) EP4255500A1 (https=)
JP (1) JP2023551911A (https=)
KR (1) KR20230128001A (https=)
AU (1) AU2021392642A1 (https=)
CA (1) CA3200192A1 (https=)
IL (1) IL303239A (https=)
MX (1) MX2023006445A (https=)
WO (1) WO2022119890A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4661916A1 (en) * 2023-02-08 2025-12-17 Ginkgo Bioworks, Inc. Gene therapy for angelman syndrome

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PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
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EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
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BR112019027692A2 (pt) 2017-06-28 2020-11-24 University Of South Florida gene ube3a modificado para uma abordagem terapêutica genética para a síndrome de angelman
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KR20210040358A (ko) * 2018-06-14 2021-04-13 오비드 테라퓨틱스 인크. 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용
WO2020132455A1 (en) * 2018-12-21 2020-06-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN114206393A (zh) * 2019-03-21 2022-03-18 Ptc医疗公司 用于治疗天使综合征的载体和方法
SG11202111375RA (en) * 2019-04-29 2021-11-29 Univ Pennsylvania Novel aav capsids and compositions containing same
US12465655B2 (en) * 2019-05-22 2025-11-11 The University Of North Carolina At Chapel Hill UBE3A genes and expression cassettes and their use
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