Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle

Exon skipping is a strategy for the treatment of Duchenne muscular dystrophy, but has variable efficacy. Here, the authors show that dystrophin restoration occurs preferentially in areas of myofiber regeneration, where antisense oligonucleotides are stored in macrophages and delivered to myoblasts a...

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Autores principales:	James S. Novak, Marshall W. Hogarth, Jessica F. Boehler, Marie Nearing, Maria C. Vila, Raul Heredia, Alyson A. Fiorillo, Aiping Zhang, Yetrib Hathout, Eric P. Hoffman, Jyoti K. Jaiswal, Kanneboyina Nagaraju, Sebahattin Cirak, Terence A. Partridge
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2017
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/2bba740be75c451786064cc88308d9ee
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spelling	oai:doaj.org-article:2bba740be75c451786064cc88308d9ee2021-12-02T14:29:49ZMyoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle10.1038/s41467-017-00924-72041-1723https://doaj.org/article/2bba740be75c451786064cc88308d9ee2017-10-01T00:00:00Zhttps://doi.org/10.1038/s41467-017-00924-7https://doaj.org/toc/2041-1723Exon skipping is a strategy for the treatment of Duchenne muscular dystrophy, but has variable efficacy. Here, the authors show that dystrophin restoration occurs preferentially in areas of myofiber regeneration, where antisense oligonucleotides are stored in macrophages and delivered to myoblasts and newly formed myotubesJames S. NovakMarshall W. HogarthJessica F. BoehlerMarie NearingMaria C. VilaRaul HerediaAlyson A. FiorilloAiping ZhangYetrib HathoutEric P. HoffmanJyoti K. JaiswalKanneboyina NagarajuSebahattin CirakTerence A. PartridgeNature PortfolioarticleScienceQENNature Communications, Vol 8, Iss 1, Pp 1-13 (2017)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q James S. Novak Marshall W. Hogarth Jessica F. Boehler Marie Nearing Maria C. Vila Raul Heredia Alyson A. Fiorillo Aiping Zhang Yetrib Hathout Eric P. Hoffman Jyoti K. Jaiswal Kanneboyina Nagaraju Sebahattin Cirak Terence A. Partridge Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
description	Exon skipping is a strategy for the treatment of Duchenne muscular dystrophy, but has variable efficacy. Here, the authors show that dystrophin restoration occurs preferentially in areas of myofiber regeneration, where antisense oligonucleotides are stored in macrophages and delivered to myoblasts and newly formed myotubes
format	article
author	James S. Novak Marshall W. Hogarth Jessica F. Boehler Marie Nearing Maria C. Vila Raul Heredia Alyson A. Fiorillo Aiping Zhang Yetrib Hathout Eric P. Hoffman Jyoti K. Jaiswal Kanneboyina Nagaraju Sebahattin Cirak Terence A. Partridge
author_facet	James S. Novak Marshall W. Hogarth Jessica F. Boehler Marie Nearing Maria C. Vila Raul Heredia Alyson A. Fiorillo Aiping Zhang Yetrib Hathout Eric P. Hoffman Jyoti K. Jaiswal Kanneboyina Nagaraju Sebahattin Cirak Terence A. Partridge
author_sort	James S. Novak
title	Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
title_short	Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
title_full	Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
title_fullStr	Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
title_full_unstemmed	Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
title_sort	myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
publisher	Nature Portfolio
publishDate	2017
url	https://doaj.org/article/2bba740be75c451786064cc88308d9ee
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Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle

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