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Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy

Cirak S, Feng L, Anthony K, et al. Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy. Molecular therapy : the journal of the American Society of Gene Therapy 2012;20:462-7   Abstract We previously conducted a proof of principle; dose escalation study in Duchenne muscular dystrophy (DMD) patients using the […]