Hightower et al., 2019 - The SINE Compound KPT-350 Blocks Dystrophic Pathologies in DMD Zebrafish and Mice. Molecular therapy : the journal of the American Society of Gene Therapy   28(1):189-201 Full text @ Mol. Ther.
Human Disease / Model Data (1 Record)
Human Disease Fish Environment Evidence Code
Duchenne muscular dystrophy dmdta222a/ta222a standard conditions