Figure 1: High-score SR protein motifs in exon 7 of SMN1 and SMN2. Figure 3 ... stems from inefficient recognition of an exonic splicing enhancer by the splicing factor SF2/ASF.

Today the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth is ...

To view the animation related to the study, please visit: SMA video "The investigational compounds used in this study represent the first orally available SMN2 splicing modifiers for SMA ...