Ontology highlight
ABSTRACT:
SUBMITTER: Garcia-Lopez A
PROVIDER: S-EPMC5966403 | biostudies-literature | 2018 May
REPOSITORIES: biostudies-literature
Nature communications 20180523 1
Modification of SMN2 exon 7 (E7) splicing is a validated therapeutic strategy against spinal muscular atrophy (SMA). However, a target-based approach to identify small-molecule E7 splicing modifiers has not been attempted, which could reveal novel therapies with improved mechanistic insight. Here, we chose as a target the stem-loop RNA structure TSL2, which overlaps with the 5' splicing site of E7. A small-molecule TSL2-binding compound, homocarbonyltopsentin (PK4C9), was identified that increas ...[more]