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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書誌詳細
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: Oxford University Press 2011
主題:
Original Articles
オンライン･アクセス: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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