Article
RNA interference improves myopathic phenotypes in mice over-expressing FSHD region gene 1 (FRG1).
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2011
Wallace Lindsay M, Garwick-Coppens Sara E, Tupler Rossella, Harper Scott Q
Abstract excerpt
Muscular dystrophies, and other diseases of muscle, arise from recessive and dominant gene mutations. Gene replacement strategies may be beneficial for the former, while gene silencing approaches may provide treatment for the latter. In the last two decades, muscle-directed gene therapies were primarily focused on treating recessive disorders. This disparity at least partly arose because feasible mechanisms to...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.