JP2024524270A5 - - Google Patents

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Publication number
JP2024524270A5
JP2024524270A5 JP2023579317A JP2023579317A JP2024524270A5 JP 2024524270 A5 JP2024524270 A5 JP 2024524270A5 JP 2023579317 A JP2023579317 A JP 2023579317A JP 2023579317 A JP2023579317 A JP 2023579317A JP 2024524270 A5 JP2024524270 A5 JP 2024524270A5
Authority
JP
Japan
Prior art keywords
muscle
sequence
vector
promoter
expression cassette
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
JP2023579317A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024524270A (ja
Filing date
Publication date
Priority claimed from GBGB2108997.4A external-priority patent/GB202108997D0/en
Application filed filed Critical
Priority claimed from PCT/GB2022/051611 external-priority patent/WO2022269269A1/en
Publication of JP2024524270A publication Critical patent/JP2024524270A/ja
Publication of JP2024524270A5 publication Critical patent/JP2024524270A5/ja
Pending legal-status Critical Current

Links

JP2023579317A 2021-06-23 2022-06-23 調節性核酸配列 Pending JP2024524270A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB2108997.4 2021-06-23
GBGB2108997.4A GB202108997D0 (en) 2021-06-23 2021-06-23 Regulatory nucleic acid sequences
GB2111317.0 2021-08-05
GB202111317 2021-08-05
PCT/GB2022/051611 WO2022269269A1 (en) 2021-06-23 2022-06-23 Regulatory nucleic acid sequences

Publications (2)

Publication Number Publication Date
JP2024524270A JP2024524270A (ja) 2024-07-05
JP2024524270A5 true JP2024524270A5 (enExample) 2025-07-02

Family

ID=82655313

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023579317A Pending JP2024524270A (ja) 2021-06-23 2022-06-23 調節性核酸配列

Country Status (8)

Country Link
US (1) US20250129381A1 (enExample)
EP (1) EP4359548A1 (enExample)
JP (1) JP2024524270A (enExample)
KR (1) KR20240023643A (enExample)
AU (1) AU2022297795A1 (enExample)
CA (1) CA3221707A1 (enExample)
IL (1) IL309630A (enExample)
WO (1) WO2022269269A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202319008D0 (en) * 2023-12-13 2024-01-24 Ucl Business Ltd AAV-mediated gene therapy of diseases caused by impaired function of GLDC

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US8106180B2 (en) 2003-08-07 2012-01-31 Whitehead Institute For Biomedical Research Methods and products for expression of micro RNAs
US20050142581A1 (en) 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
WO2005056761A2 (en) 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
KR100614827B1 (ko) 2004-05-06 2006-08-25 재단법인서울대학교산학협력재단 양방향 은닉 마코프 모델을 이용한 완숙한마이크로알엔에이 위치예측방법 및 이를 구현하기 위한컴퓨터 프로그램을 기록한 저장매체
AU2015208247B2 (en) * 2014-01-21 2021-05-27 Universiteit Gent Muscle-specific nucleic acid regulatory elements and methods and use thereof
AU2020392243A1 (en) * 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
EP4081643A1 (en) * 2019-12-24 2022-11-02 Asklepios Biopharmaceutical, Inc. Regulatory nucleic acid sequences
CN116406297A (zh) * 2020-08-05 2023-07-07 阿斯克肋匹奥生物制药公司 治疗心脏疾患和充血性心力衰竭以及施用aav载体的方法
US20230374542A1 (en) * 2020-10-07 2023-11-23 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 (lgmd21)

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