Translational and Regulatory Challenges for Exon Skipping Therapies

Aartsma-Rus A, Ferlini A, Goemans N, et al. Translational and regulatory challenges for exon skipping therapies. Human gene therapy 2014;25:885-92   Abstract Several translational challenges are currently impeding the therapeutic development of antisense-mediated exon skipping approaches for rare diseases. Some of these are inherent to developing therapies for rare diseases, such as small patient numbers […]