509 Genetic Correction of Duchenne Muscular Dystrophy by Multiplex CRISPR/Cas9 Based Gene Editing Molecular Therapy Volume 22, Supplement 1, May 2014 Copyright © The American Society of Gene & Cell Th[.]
THERAPIES FOR MYOGENIC DISORDERS of transduced cells necessary to achieve functional anion transport correction Due to the anticipated transient episomal expression and immune responses to first generation Ad vectors, we limited these studies to short-term (