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Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

✍ Scribed by Cirak, Sebahattin; Feng, Lucy; Anthony, Karen; Arechavala-Gomeza, Virginia; Torelli, Silvia; Sewry, Caroline; Morgan, Jennifer E; Muntoni, Francesco

Book ID
118052982
Publisher
Nature Publishing Group
Year
2012
Tongue
English
Weight
350 KB
Volume
20
Category
Article
ISSN
1525-0016

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Out of three mutations in the dystrophin gene that cause Duchenne muscular dystrophy (DMD), the most common, serious childhood muscle wasting disease, two are genomic deletions of one or more exons that disrupt the reading frame. Specific removal of an exon flanking a genomic deletion using antisens