JP2021512072A5 - - Google Patents

Info

Publication number
JP2021512072A5
JP2021512072A5 JP2020540722A JP2020540722A JP2021512072A5 JP 2021512072 A5 JP2021512072 A5 JP 2021512072A5 JP 2020540722 A JP2020540722 A JP 2020540722A JP 2020540722 A JP2020540722 A JP 2020540722A JP 2021512072 A5 JP2021512072 A5 JP 2021512072A5
Authority
JP
Japan
Prior art keywords
muscle
composition
item
aav
sarcoglycan
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.)
Granted
Application number
JP2020540722A
Other languages
English (en)
Japanese (ja)
Other versions
JP7361701B2 (ja
JP2021512072A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/015779 external-priority patent/WO2019152474A1/en
Publication of JP2021512072A publication Critical patent/JP2021512072A/ja
Publication of JP2021512072A5 publication Critical patent/JP2021512072A5/ja
Priority to JP2023171950A priority Critical patent/JP7752157B2/ja
Application granted granted Critical
Publication of JP7361701B2 publication Critical patent/JP7361701B2/ja
Priority to JP2025121245A priority patent/JP2025162557A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2020540722A 2018-01-31 2019-01-30 肢帯型筋ジストロフィー2c型のための遺伝子治療 Active JP7361701B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023171950A JP7752157B2 (ja) 2018-01-31 2023-10-03 肢帯型筋ジストロフィー2c型のための遺伝子治療
JP2025121245A JP2025162557A (ja) 2018-01-31 2025-07-18 肢帯型筋ジストロフィー2c型のための遺伝子治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862624616P 2018-01-31 2018-01-31
US62/624,616 2018-01-31
PCT/US2019/015779 WO2019152474A1 (en) 2018-01-31 2019-01-30 Gene therapy for limb-girdle muscular dystrophy type 2c

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023171950A Division JP7752157B2 (ja) 2018-01-31 2023-10-03 肢帯型筋ジストロフィー2c型のための遺伝子治療

Publications (3)

Publication Number Publication Date
JP2021512072A JP2021512072A (ja) 2021-05-13
JP2021512072A5 true JP2021512072A5 (https=) 2022-02-07
JP7361701B2 JP7361701B2 (ja) 2023-10-16

Family

ID=67478478

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2020540722A Active JP7361701B2 (ja) 2018-01-31 2019-01-30 肢帯型筋ジストロフィー2c型のための遺伝子治療
JP2023171950A Active JP7752157B2 (ja) 2018-01-31 2023-10-03 肢帯型筋ジストロフィー2c型のための遺伝子治療
JP2025121245A Pending JP2025162557A (ja) 2018-01-31 2025-07-18 肢帯型筋ジストロフィー2c型のための遺伝子治療

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2023171950A Active JP7752157B2 (ja) 2018-01-31 2023-10-03 肢帯型筋ジストロフィー2c型のための遺伝子治療
JP2025121245A Pending JP2025162557A (ja) 2018-01-31 2025-07-18 肢帯型筋ジストロフィー2c型のための遺伝子治療

Country Status (18)

Country Link
US (2) US12263230B2 (https=)
EP (1) EP3746100A4 (https=)
JP (3) JP7361701B2 (https=)
KR (2) KR20250099758A (https=)
CN (2) CN111818946B (https=)
AR (1) AR114350A1 (https=)
AU (2) AU2019216257B2 (https=)
BR (1) BR112020015173A2 (https=)
CA (1) CA3089080A1 (https=)
CL (2) CL2020001948A1 (https=)
CO (1) CO2020010270A2 (https=)
EA (1) EA202091739A1 (https=)
IL (2) IL320714A (https=)
MX (1) MX2020007876A (https=)
MY (1) MY209273A (https=)
SG (1) SG11202006722RA (https=)
TW (1) TWI815856B (https=)
WO (1) WO2019152474A1 (https=)

Families Citing this family (19)

* 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
KR102874891B1 (ko) 2018-01-30 2025-10-21 애프니메드, 인코포레이티드 (델라웨어) 수면 무호흡증을 치료하기 위한 방법 및 조성물
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
WO2021087007A1 (en) * 2019-10-28 2021-05-06 University Of Florida Research Foundation, Incorporated Gene therapy vectors
WO2021126880A1 (en) * 2019-12-16 2021-06-24 Research Institute At Nationwide Children's Hospital Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc)
IL299094A (en) * 2020-06-15 2023-02-01 Res Inst Nationwide Childrens Hospital Administration of an adeno-associated virus vector for muscular dystrophies
US20230256117A1 (en) * 2020-06-19 2023-08-17 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
CN113952472A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
TWI887479B (zh) 2020-09-08 2025-06-21 美商薩羅塔治療公司 表現γ-肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療
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
EP4186919A1 (en) * 2021-11-30 2023-05-31 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
EP4198134A1 (en) 2021-12-16 2023-06-21 Genethon Gamma-sarcoglycan gene transfer increase using modified itr sequences
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders

Family Cites Families (105)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0127839B1 (en) 1983-05-27 1992-07-15 THE TEXAS A&M UNIVERSITY SYSTEM Method for producing a recombinant baculovirus expression vector
ZA848495B (en) 1984-01-31 1985-09-25 Idaho Res Found Production of polypeptides in insect cells
US5449616A (en) 1990-05-23 1995-09-12 University Of Iowa Research Foundation Nucleic acid encoding dystrophin-associated protein
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
WO1995003392A1 (en) 1993-07-23 1995-02-02 Unichema Chemie Bv Process for producing transparent soap material
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5672694A (en) 1995-10-24 1997-09-30 University Of Iowa Research Foundation β-sarcoglycan nucleic acid sequence, and nucleic acid probes
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP2001500497A (ja) 1996-09-06 2001-01-16 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 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法
US5843078A (en) 1997-07-01 1998-12-01 Sharkey; Hugh R. Radio frequency device for resurfacing skin and method
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999043360A1 (en) 1998-02-26 1999-09-02 The Trustees Of The University Of Pennsylvania Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex
US6262035B1 (en) * 1998-10-01 2001-07-17 University Of Iowa Research Foundation Gene replacement therapy for muscular dystrophy
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6632800B1 (en) 1999-08-17 2003-10-14 Mayo Foundation For Medical Education And Research System for monitoring the expression of transgenes
JP4520569B2 (ja) 2000-02-18 2010-08-04 照彦 豊岡 拡張型心筋症の遺伝子治療剤
US7056502B2 (en) * 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US20040126762A1 (en) 2002-12-17 2004-07-01 Morris David W. Novel compositions and methods in cancer
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
AU2003212708A1 (en) 2002-03-05 2003-09-16 Stichting Voor De Technische Wetenschappen Baculovirus expression system
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
US20030225260A1 (en) 2002-04-30 2003-12-04 Snyder Richard O. Production of recombinant AAV virions
BRPI0511764B8 (pt) 2004-06-01 2021-05-25 Avigen Inc método de prevenção de agregação de vírions de vírus adeno-associado recombinante (raav) em uma preparação purificada de virions raav
US7972593B2 (en) 2004-06-10 2011-07-05 Saint Louis University Delivery of therapeutic agents to the bone
US20090054823A1 (en) 2004-09-30 2009-02-26 The Trustees Of The University Of Pennsylvania Perfusion circuit and use therein in targeted delivery of macromolecules
JP2006121961A (ja) 2004-10-28 2006-05-18 Univ Of Tokushima 顎骨骨幹異形成症gddの原因遺伝子gdd1とその用途
US7883858B2 (en) 2005-01-27 2011-02-08 Institute For Systems Biology Methods for identifying and monitoring drug side effects
US7788045B2 (en) 2005-09-01 2010-08-31 Meditasks, Llc Systems and method for homeostatic blood states
EP1938104A2 (en) 2005-10-17 2008-07-02 Institute for Systems Biology Tissue-and serum-derived glycoproteins and methods of their use
WO2007049095A1 (en) 2005-10-25 2007-05-03 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
EP2052088A2 (en) 2006-08-02 2009-04-29 Genizon Biosciences Genemap of the human genes associated with psoriasis
AU2007284651B2 (en) 2006-08-09 2014-03-20 Institute For Systems Biology Organ-specific proteins and methods of their use
EP2087098A4 (en) 2006-11-09 2010-03-31 Univ Johns Hopkins DETERMINATION OF CARDIOMYOCYTES ADULT MAMMALS IN HEART STEM CELLS
CN101711164B (zh) 2007-01-18 2014-06-04 密苏里-哥伦比亚大学 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因
FR2919305B1 (fr) 2007-07-26 2009-09-18 Genethon Ass Loi De 1901 Vecteurs viraux adeno-associes pour l'expression de la dysferline.
GB0715087D0 (en) 2007-08-03 2007-09-12 Summit Corp Plc Drug combinations for the treatment of duchenne muscular dystrophy
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
JP5634272B2 (ja) 2008-03-14 2014-12-03 ヒューマンザイム リミテッド ヒト細胞発現系を用いる真正ヒトタンパク質の組換え生産
US20090280103A1 (en) 2008-04-04 2009-11-12 Martin Flueck Regulation of muscle repair
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
US20100026655A1 (en) 2008-07-31 2010-02-04 Avago Technologies Ecbu Ip (Singapore) Pte. Ltd. Capacitive Touchscreen or Touchpad for Finger or Stylus
US8729041B2 (en) 2008-12-03 2014-05-20 The Johns Hopkins University Compositions and methods for treating hepatic neoplasia
US20110023139A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20100247495A1 (en) * 2009-03-30 2010-09-30 Tom Ichim Treatment of Muscular Dystrophy
WO2011044138A1 (en) 2009-10-05 2011-04-14 Catabasis Pharmaceuticals, Inc. Lipoic acid acylated salicylate derivatives and their uses
KR101841752B1 (ko) 2009-11-03 2018-05-04 더 리전트 오브 더 유니버시티 오브 캘리포니아 저온 수용액 내에서 성장한 다중 표면들 상의 산화 아연 층들에 의해 커버되는 고휘도 발광 다이오드
FR2962041B1 (fr) 2010-07-01 2012-07-27 Genethon Inhibiteurs de la calpaine 3 pour le traitement de dystrophies musculaires et de cardiomyopathies
EP2736539B1 (en) 2011-07-25 2017-08-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
MX367100B (es) 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
US9254311B2 (en) * 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2014037526A1 (en) 2012-09-07 2014-03-13 INSERM (Institut National de la Santé et de la Recherche Médicale) In vitro genetic diagnostic of inherited neuromuscular disorders
EP2900821B1 (en) 2012-09-25 2020-04-01 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
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
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
FR3004463A1 (fr) 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
ES2666322T3 (es) 2013-10-08 2018-05-04 Ystem S.R.L. Composiciones farmacéuticas para el tratamiento de trastornos musculares
US9850497B2 (en) 2013-11-04 2017-12-26 Regents Of The University Of Minnesota Gene targeting methods and tools
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
PL3097197T3 (pl) 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
DE102014207498A1 (de) 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
JP6832280B2 (ja) 2015-01-16 2021-02-24 ユニバーシティ オブ ワシントンUniversity of Washington 新規のマイクロジストロフィンおよび使用の関連する方法
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
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
ES2977730T3 (es) 2016-04-15 2024-08-29 Res Inst Nationwide Childrens Hospital Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular
MX2018012537A (es) 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
LT3442600T (lt) 2016-04-15 2024-05-27 Research Institute At Nationwide Children's Hospital Su adeno virusu susijusio viruso vektoriaus iš β-sarkoglikano ir mikro- rnr-29 pristatymas ir raumenų distrofijos gydymas
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
CA2971303C (en) 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
JP7676111B2 (ja) 2017-03-17 2025-05-14 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達
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
MA50836A (fr) 2017-10-18 2020-08-26 Res Inst Nationwide Childrens Hospital Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire
US11926653B2 (en) 2017-10-20 2024-03-12 Research Institute At Nationwide Children's Hospital Methods and materials for NT-3 gene therapy
WO2019118806A1 (en) 2017-12-14 2019-06-20 Solid Biosciences Inc. Non-viral production and delivery of genes
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
US20210139550A1 (en) 2018-04-03 2021-05-13 Curators Of The University Of Missouri Hinges 1 and/or 4 modified dystrophins for dystrophinopathy therapy
US12152242B2 (en) 2018-04-23 2024-11-26 The Curators Of The University Of Missouri CRISPR therapy
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
WO2020123645A1 (en) 2018-12-12 2020-06-18 Solid Biosciences Inc. Combination therapy for treating muscular dystrophy
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
IL299094A (en) 2020-06-15 2023-02-01 Res Inst Nationwide Childrens Hospital Administration of an adeno-associated virus vector for muscular dystrophies
JP2023534127A (ja) 2020-06-17 2023-08-08 アクセルロン ファーマ インコーポレイテッド ActRII-ALK4アンタゴニストおよび心不全を処置する方法

Similar Documents

Publication Publication Date Title
JP2021512072A5 (https=)
JP2025162557A5 (https=)
AU2023200175B2 (en) Recombinant Adeno-Associated Virus Delivery Of Exon 2-Targeted U7snRNA Polynucleotide Constructs
IL275880B1 (en) Gene therapy for limb-girdle muscular dystrophy type 2c
JP2019513393A5 (https=)
JP2020513811A5 (https=)
JP2019513389A5 (https=)
JP2019513399A5 (https=)
JP2019513779A5 (https=)
JP7646362B2 (ja) 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクターデリバリー
JP7189771B2 (ja) 筋ジストロフィーを治療するためのマイクロrna-29およびマイクロジストロフィンのアデノ随伴ウイルスベクター送達
JP2020510447A5 (https=)
RU2018116076A (ru) Модифицированные гены атаксии фридрейха и векторы для генной терапии
JP2021500352A5 (https=)
JP2022551986A5 (https=)
JP2021528983A5 (https=)
JP2020533959A5 (https=)
JP2021512649A5 (https=)
JP2024159800A (ja) 筋肉発現のためのハイブリッドプロモーター
JP2017529395A5 (https=)
JP2023537358A5 (https=)
JP2023059858A5 (https=)
CN114466682A (zh) 用于治疗基于肌营养不良蛋白的肌病的靶向外显子44的核酸和包含所述核酸的重组腺相关病毒
CN116917492A (zh) 用于抑制外周髓鞘蛋白-22的表达的产物和方法
JP2022545378A5 (https=)