Article
Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration.
Human molecular genetics - 1 Dec 2015
Roberts Thomas C, Johansson Henrik J, McClorey Graham, Godfrey Caroline, Blomberg K Emelie M, Coursindel Thibault, Gait Michael J, Smith C I Edvard, Lehtiö Janne, El Andaloussi Samir, Wood Matthew J A
Abstract excerpt
Duchenne muscular dystrophy (DMD) is a classical monogenic disorder, a model disease for genomic studies and a priority candidate for regenerative medicine and gene therapy. Although the genetic cause of DMD is well known, the molecular pathogenesis of disease and the response to therapy are incompletely understood. Here, we describe analyses of protein, mRNA and microRNA expression in the tibialis anterior of...
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