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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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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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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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AAV-based gene therapies for the muscular dystrophies

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

2019

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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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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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Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review

BioDrugs : clinical immunotherapeutics, biopharmaceuticals, and gene therapy, 2024-01, Vol.38 (1), p.95-119

2024

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Viral vector-mediated gene therapies

Current opinion in neurology, 2015-10, Vol.28 (5), p.522-527

2015

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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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Surrogate gene therapy for muscular dystrophy

Nature medicine, 2019-10, Vol.25 (10), p.1473-1474

2019

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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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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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Cidofovir Diphosphate Inhibits Adenovirus 5 DNA Polymerase via both Nonobligate Chain Termination and Direct Inhibition, and Polymerase Mutations Confer Cidofovir Resistance on Intact Virus

Antimicrobial agents and chemotherapy, 2019-01, Vol.63 (1)

2019

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Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins

Human molecular genetics, 2018-06, Vol.27 (12), p.2090-2100

2018

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Progress and prospects of gene therapy clinical trials for the muscular dystrophies

Human molecular genetics, 2016-04, Vol.25 (R1), p.R9-R17

2016

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Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model

Molecular therapy, 2022-06, Vol.30 (6), p.2176-2185

2022

Volltextzugriff (PDF)

AAV6 Vector Production and Purification for Muscle Gene Therapy

Methods in molecular biology (Clifton, N.J.), 2018, Vol.1687, p.257

2018

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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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Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery

Cell, 2023-05, Vol.186 (10), p.2062-2077.e17

2023

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Split intein-mediated protein trans-splicing to express large dystrophins

Nature (London), 2024-08, Vol.632 (8023), p.192-200

2024

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The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins

PLoS genetics, 2010-05, Vol.6 (5), p.e1000958-e1000958

2010

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A Live Attenuated Influenza Vaccine Elicits Enhanced Heterologous Protection When the Internal Genes of the Vaccine Are Matched to Those of the Challenge Virus

Journal of virology, 2020-01, Vol.94 (4)

2020

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Progress toward Gene Therapy for Duchenne Muscular Dystrophy

Cas9 immunity creates challenges for CRISPR gene editing therapies

A Boost for Muscle with Gene Therapy

AAV-based gene therapies for the muscular dystrophies

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

Gene and cell-mediated therapies for muscular dystrophy

Recombinant Adeno-associated Virus Transduction and Integration

Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review

Viral vector-mediated gene therapies

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

Surrogate gene therapy for muscular dystrophy

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

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

Cidofovir Diphosphate Inhibits Adenovirus 5 DNA Polymerase via both Nonobligate Chain Termination and Direct Inhibition, and Polymerase Mutations Confer Cidofovir Resistance on Intact Virus

Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins

Progress and prospects of gene therapy clinical trials for the muscular dystrophies

Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model

AAV6 Vector Production and Purification for Muscle Gene Therapy

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

Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery

Split intein-mediated protein trans-splicing to express large dystrophins

The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins

A Live Attenuated Influenza Vaccine Elicits Enhanced Heterologous Protection When the Internal Genes of the Vaccine Are Matched to Those of the Challenge Virus

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