Molecule Boosts Antisense Therapy For Muscular Dystrophy (1 of 1) (IMAGE)
Caption
Dantrolene improves antisense oligonucleotide mediated exon skipping efficiency. Induced exon skipping is a promising therapy for Duchenne muscular dystrophy (DMD), but current clinical trials rescue variable amounts of dystrophin protein. The authors identify a FDA approved drug, dantrolene, which enhances antisense oligonucleotide (AO) directed exon skipping in a DMD mouse model, increases dystrophin protein expression, and improves muscle function (hang time) in mice. In this artistic rendering the three dimensional electron density of dantrolene blends into the density profile of a RNA molecule, and onto a dystrophin protein labeling of a mouse muscle cross section. This image relates to a paper that appeared in the December 12, 2012, issue of Science Translational Medicine, published by AAAS. The paper, by G.C. Kendall at David Geffen School of Medicine, University of California, Los Angeles in Los Angeles, Calif., and colleagues was titled, "Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy."
Credit
Image courtesy of G.C. Kendall; input from M.C. Miceli and S.F. Nelson; original images provided by E.I. Mokhonova and M.J. Spencer
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