RT Journal Article SR Electronic T1 Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road? JF Science Translational Medicine FD American Association for the Advancement of Science SP 230fs14 OP 230fs14 DO 10.1126/scitranslmed.3008873 VO 6 IS 230 A1 Hoffman, Eric P. A1 McNally, Elizabeth M. YR 2014 UL http://stm.sciencemag.org/content/6/230/230fs14.abstract AB Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.