Genome editing shows promise in an in vivo model of duchenne muscular dystrophy

Access through your institution Buy or subscribe CRISPR–Cas9-based genome editing can restore dystrophin protein expression and improve muscle structure and function in the _mdx_ mouse model

of Duchenne muscular dystrophy (DMD), according to three reports recently published in _Science_. Most cases of DMD are caused by frameshift mutations in the dystrophin (_DMD_) gene that

lead to the production of a truncated, non-functional form of the dystrophin protein. The genome editing strategy is based on the premise that excision of one or more exons of the mutant

gene can restore the DMD reading frame, enabling an internally truncated but functional protein to be generated. This is a preview of subscription content, access via your institution ACCESS

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institutional subscriptions * Read our FAQs * Contact customer support REFERENCES * Nelson, C. E. _ et al_. _In vivo_ genome editing improves muscle function in a mouse model of Duchenne

muscular dystrophy. _Science_ http://dx.doi.org/10.1126/science.aad5143 * Tabebordbar, M. _ et al_. _In vivo_ gene editing in dystrophic mouse muscle and muscle stem cells. _Science_

http://dx.doi.org/10.1126/science.aad5177 * Long, C. _ et al_. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. _Science_

http://dx.doi.org/10.1126/science.aad5725 Download references Authors * Heather Wood View author publications You can also search for this author inPubMed Google Scholar RELATED LINKS

RELATED LINKS RELATED LINKS IN NATURE RESEARCH Ousterout, D. G. _ et al_. Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular

dystrophy. _Nat. Commun._ 6, 6244 (2015) RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Wood, H. Genome editing shows promise in an _in vivo_ model of

Duchenne muscular dystrophy. _Nat Rev Neurol_ 12, 63 (2016). https://doi.org/10.1038/nrneurol.2016.4 Download citation * Published: 18 January 2016 * Issue Date: February 2016 * DOI:

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