Publication authors

Annemieke Aartsma-Rus

Journal

Nucleic Acid Therapeutics, volume 24, issue 1,

Publication date

February 2014

Abstract
While antisense-mediated splicing modulation is advanced furthest for Duchenne muscular dystrophy, the approach has therapeutic potential for a variety of other diseases, being in phase 2 clinical trials for spinal muscular atrophy and in preclinical development for numerous other diseases (van Roon-Mom and Aartsma-Rus, 2012).

DOI link

10.1089/nat.2014.1500