JP2021520196A5 - - Google Patents

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Publication number
JP2021520196A5
JP2021520196A5 JP2020554102A JP2020554102A JP2021520196A5 JP 2021520196 A5 JP2021520196 A5 JP 2021520196A5 JP 2020554102 A JP2020554102 A JP 2020554102A JP 2020554102 A JP2020554102 A JP 2020554102A JP 2021520196 A5 JP2021520196 A5 JP 2021520196A5
Authority
JP
Japan
Prior art keywords
adeno
associated virus
avv
muscular dystrophy
vector
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
JP2020554102A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021520196A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/025496 external-priority patent/WO2019195362A1/en
Publication of JP2021520196A publication Critical patent/JP2021520196A/ja
Publication of JP2021520196A5 publication Critical patent/JP2021520196A5/ja
Pending legal-status Critical Current

Links

JP2020554102A 2018-04-03 2019-04-03 ジストロフィン異常症(dystrophinopathy)治療のためのヒンジ1および/または4の改変ジストロフィン Pending JP2021520196A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862651772P 2018-04-03 2018-04-03
US62/651,772 2018-04-03
PCT/US2019/025496 WO2019195362A1 (en) 2018-04-03 2019-04-03 Hinges 1 and/or 4 modified dystrophins for dystrophinopathy therapy

Publications (2)

Publication Number Publication Date
JP2021520196A JP2021520196A (ja) 2021-08-19
JP2021520196A5 true JP2021520196A5 (https=) 2022-04-05

Family

ID=68101401

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020554102A Pending JP2021520196A (ja) 2018-04-03 2019-04-03 ジストロフィン異常症(dystrophinopathy)治療のためのヒンジ1および/または4の改変ジストロフィン

Country Status (5)

Country Link
US (1) US20210139550A1 (https=)
EP (1) EP3773605A4 (https=)
JP (1) JP2021520196A (https=)
CN (1) CN111971050A (https=)
WO (1) WO2019195362A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102877920B1 (ko) 2015-11-16 2025-10-30 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
EP3596112A2 (en) 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
AR114350A1 (es) 2018-01-31 2020-08-26 Res Institute At Nationwide Children’S Hospital Terapia genética para la distrofia muscular de cinturas del tipo 2c
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
AU2020229340A1 (en) 2019-02-26 2021-09-16 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy
RS65421B1 (sr) 2019-08-21 2024-05-31 Res Inst Nationwide Childrens Hospital Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije
IL297753A (en) * 2020-04-29 2022-12-01 Bristol Myers Squibb Co Minimized dystrophins with spectrin fusion domains and uses thereof
IL299094A (en) 2020-06-15 2023-02-01 Res Inst Nationwide Childrens Hospital Administration of an adeno-associated virus vector for muscular dystrophies
WO2022029543A1 (en) * 2020-08-06 2022-02-10 Intas Pharmaceuticals Ltd. Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
EP4108263A3 (en) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7771993B2 (en) * 2004-01-23 2010-08-10 The Trustees Of The University Of Pennsylvania Microutrophin and uses thereof
US9624282B2 (en) * 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
WO2017223128A1 (en) * 2016-06-21 2017-12-28 The Curators Of The University Of Missouri Modified dystrophin proteins

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