Duchenne Muscular Dystrophy
New Duchenne Muscular Dystrophy Findings from University of Oxford Described
Published in Gene Therapy Weekly, August 25th, 2011
"Antisense oligonucleotide (AO)-mediated exon skipping has been shown to restore functional dystrophin in mdx mice and DMD patients treated intramuscularly in two recent phase 1 clinical trials. Critical to the therapeutic success of AO-based treatment will be the ability to deliver AOs...
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Source: Gene Therapy Weekly (2011-08-25)
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