Fig. 1
From: Stathmin 2 is a potential treatment target for TDP-43 proteinopathy in amyotrophic lateral sclerosis
TDP-43 binds directly to STMN2 pre-mRNA to guarantee normal splicing of STMN2 mRNA. Pathogenic (reduced) TDP-43 drives premature polyadenylation and aberrant splicing by steric inhibition in the first intron of the STMN2 pre-mRNA, producing a non-functional mRNA. Using dCasRx or antisense oligonucleotides (ASOs) to target the first intron of the STMN2 pre-mRNA can efficiently restore STMN2 level and axonal regeneration in TDP-43 proteinopathy. Although current studies have provided promising results, animal models are required to confirm the efficiency and safety before clinic trials