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Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration

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 incompl...

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Bibliografische gegevens
Gepubliceerd in:Hum Mol Genet
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Oxford University Press 2015
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4634378/
https://ncbi.nlm.nih.gov/pubmed/26385637
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv381
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