A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping.

Duchenne muscular dystrophy (DMD), which afflicts 1 in 3500 boys, is one of the most common genetic disorders of children. This fatal degenerative condition is caused by an absence or deficiency of dystrophin in striated muscle. Most affected patients have inherited or spontaneous deletions in the d...

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Bibliographic Details
Main Authors: Gemma L Walmsley, Virginia Arechavala-Gomeza, Marta Fernandez-Fuente, Margaret M Burke, Nicole Nagel, Angela Holder, Rachael Stanley, Kate Chandler, Stanley L Marks, Francesco Muntoni, G Diane Shelton, Richard J Piercy
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2010-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC2800183?pdf=render