intravenous exon skipping

A trial of AVI4658, an experimental exon skipping construct for DMD, resulted in dystrophin production and appears safe

posted on April 15, 2010 - 3:57pm
AVI4658, an experimental treatment for patients with Duchenne muscular dystrophy (DMD) caused by certain mutations in the gene for the muscle protein dystrophin, has shown promising results when delivered intravenously to 19 trial participants.

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