Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

Duchenne muscular dystrophy is caused by mutations in the DMD gene that disrupt the open reading frame and prevent the full translation of its protein product, dystrophin. Restoration of the open reading frame and dystrophin production can be achieved by exon skipping using antisense oligonucleotide...

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Bibliographic Details
Main Authors: Anthony, Karen, Cirak, Sebahattin, Torelli, Silvia, Tasca, Giorgio, Feng, Lucy, Arechavala-Gomeza, Virginia, Armaroli, Annarita, Guglieri, Michela, Straathof, Chiara S., Verschuuren, Jan J., Aartsma-Rus, Annemieke, Helderman-van den Enden, Paula, Bushby, Katherine, Straub, Volker, Sewry, Caroline, Ferlini, Alessandra, Ricci, Enzo, Morgan, Jennifer E., Muntoni, Francesco
Format: Artigo
Language:Inglês
Published: Oxford University Press 2011
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Original Articles
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3235564/
https://ncbi.nlm.nih.gov/pubmed/22102647
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/brain/awr291
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