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Cas9 immunity creates challenges for CRISPR gene editing therapies

Nature communications, 2018-08, Vol.9 (1), p.3497-3, Article 3497

2018

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Progress toward Gene Therapy for Duchenne Muscular Dystrophy

Molecular therapy, 2017-05, Vol.25 (5), p.1125-1131

2017

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Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

Nature communications, 2017-02, Vol.8 (1), p.14454-10, Article 14454

2017

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High levels of AAV vector integration into CRISPR-induced DNA breaks

Nature communications, 2019-09, Vol.10 (1), p.4439-11, Article 4439

2019

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Gene and cell-mediated therapies for muscular dystrophy

Muscle & nerve, 2013-05, Vol.47 (5), p.649-663

2013

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Recombinant Adeno-associated Virus Transduction and Integration

Molecular therapy, 2008-07, Vol.16 (7), p.1189-1199

2008

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Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy

The Journal of clinical investigation, 2009-03, Vol.119 (3), p.624-635

2009

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Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials

Human gene therapy, 2023-05, Vol.34 (9-10), p.404-415

2023

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Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells

Nature cell biology, 2007-03, Vol.9 (3), p.255-267

2007

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Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects

PLoS genetics, 2020-11, Vol.16 (11), p.e1009179-e1009179

2020

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Follistatin-mediated skeletal muscle hypertrophy is regulated by Smad3 and mTOR independently of myostatin

The Journal of cell biology, 2012-06, Vol.197 (7), p.997-1008

2012

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Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16-17

Human molecular genetics, 2018-09, Vol.27 (17), p.2978-2985

2018

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An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

Nature communications, 2013, Vol.4 (1), p.1549-1549, Article 1549

2013

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Sarcolemma-localized nNOS is required to maintain activity after mild exercise

Nature, 2008-11, Vol.456 (7221), p.511-515

2008

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Prevention of muscle aging by myofiber-associated satellite cell transplantation

Science translational medicine, 2010-11, Vol.2 (57), p.57ra83

2010

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Non-invasive tracking of disease progression in young dystrophic muscles using multi-parametric MRI at 14T

PloS one, 2018-10, Vol.13 (10), p.e0206323-e0206323

2018

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rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice

Nature medicine, 2006-07, Vol.12 (7), p.787-789

2006

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Gene therapy for Duchenne muscular dystrophy

Expert opinion on orphan drugs, 2015-11, Vol.3 (11), p.1255-1266

2015

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A Boost for Muscle with Gene Therapy

The New England journal of medicine, 2022-03, Vol.386 (12), p.1184-1186

2022

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Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy

Proceedings of the National Academy of Sciences - PNAS, 2018-09, Vol.115 (39), p.E9182-E9191

2018

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Multi-parametric MRI at 14T for muscular dystrophy mice treated with AAV vector-mediated gene therapy

PloS one, 2015-04, Vol.10 (4), p.e0124914-e0124914

2015

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Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies

Molecular therapy, 2012-08, Vol.20 (8), p.1501-1507

2012

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Microutrophin Delivery Through rAAV6 Increases Lifespan and Improves Muscle Function in Dystrophic Dystrophin/Utrophin-deficient Mice

Molecular therapy, 2008-09, Vol.16 (9), p.1539-1545

2008

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AAV-based gene therapies for the muscular dystrophies

Human molecular genetics, 2019-10, Vol.28 (R1), p.R102-R107

2019

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A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

Molecular therapy. Methods & clinical development, 2017-09, Vol.6 (C), p.216-230

2017

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Cas9 immunity creates challenges for CRISPR gene editing therapies

Progress toward Gene Therapy for Duchenne Muscular Dystrophy

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

High levels of AAV vector integration into CRISPR-induced DNA breaks

Gene and cell-mediated therapies for muscular dystrophy

Recombinant Adeno-associated Virus Transduction and Integration

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy

Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials

Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells

Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects

Follistatin-mediated skeletal muscle hypertrophy is regulated by Smad3 and mTOR independently of myostatin

Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16-17

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

Sarcolemma-localized nNOS is required to maintain activity after mild exercise

Prevention of muscle aging by myofiber-associated satellite cell transplantation

Non-invasive tracking of disease progression in young dystrophic muscles using multi-parametric MRI at 14T

rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice

Gene therapy for Duchenne muscular dystrophy

A Boost for Muscle with Gene Therapy

Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy

Multi-parametric MRI at 14T for muscular dystrophy mice treated with AAV vector-mediated gene therapy

Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies

Microutrophin Delivery Through rAAV6 Increases Lifespan and Improves Muscle Function in Dystrophic Dystrophin/Utrophin-deficient Mice

AAV-based gene therapies for the muscular dystrophies

A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

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