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

CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrophin expression in skeletal and cardiac muscle in m...

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Autores principales:	Niclas E. Bengtsson, John K. Hall, Guy L. Odom, Michael P. Phelps, Colin R. Andrus, R. David Hawkins, Stephen D. Hauschka, Joel R. Chamberlain, Jeffrey S. Chamberlain
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2017
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/42b84f4d2d7e485cb3c0f8f3e7eaf353
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spelling	oai:doaj.org-article:42b84f4d2d7e485cb3c0f8f3e7eaf3532021-12-02T15:38:43ZMuscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy10.1038/ncomms144542041-1723https://doaj.org/article/42b84f4d2d7e485cb3c0f8f3e7eaf3532017-02-01T00:00:00Zhttps://doi.org/10.1038/ncomms14454https://doaj.org/toc/2041-1723CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrophin expression in skeletal and cardiac muscle in mice.Niclas E. BengtssonJohn K. HallGuy L. OdomMichael P. PhelpsColin R. AndrusR. David HawkinsStephen D. HauschkaJoel R. ChamberlainJeffrey S. ChamberlainNature PortfolioarticleScienceQENNature Communications, Vol 8, Iss 1, Pp 1-10 (2017)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Niclas E. Bengtsson John K. Hall Guy L. Odom Michael P. Phelps Colin R. Andrus R. David Hawkins Stephen D. Hauschka Joel R. Chamberlain Jeffrey S. Chamberlain Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
description	CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrophin expression in skeletal and cardiac muscle in mice.
format	article
author	Niclas E. Bengtsson John K. Hall Guy L. Odom Michael P. Phelps Colin R. Andrus R. David Hawkins Stephen D. Hauschka Joel R. Chamberlain Jeffrey S. Chamberlain
author_facet	Niclas E. Bengtsson John K. Hall Guy L. Odom Michael P. Phelps Colin R. Andrus R. David Hawkins Stephen D. Hauschka Joel R. Chamberlain Jeffrey S. Chamberlain
author_sort	Niclas E. Bengtsson
title	Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
title_short	Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
title_full	Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
title_fullStr	Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
title_full_unstemmed	Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
title_sort	muscle-specific crispr/cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for duchenne muscular dystrophy
publisher	Nature Portfolio
publishDate	2017
url	https://doaj.org/article/42b84f4d2d7e485cb3c0f8f3e7eaf353
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Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

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