Targeting STMN2 Splicing to Treat ALS

Today’s TransMission is a SNiP (Small News in Pieces) about stathmin-2, which is encoded by the STMN2 gene. No, this is not a pun on a Jason Statham action movie. STMN2 is a potential drug target for amyotrophic lateral sclerosis (ALS). In this SNiP, we highlight how Baughn & Melamed et al. modulate splicing of STMN2 mRNA with antisense oligonucleotide- and CRISPR-based approaches. The strategies and tools introduced in their study will be useful in the development of gene therapies to treat ALS and related neurodegenerative diseases.