Non-Coding RNAs and Paracrine Signalling in Skeletal Muscle Cells: Implications for ALS treatment

Amyotrophic Lateral Sclerosis (ALS) is a disease that affects skeletal muscles. Despite its high relevance, it has not been deeply studied from a genetic and molecular perspective. There are no cures, only treatments that can slow the progression of the disease. Recently, some gene therapy treatments have been developed using non-coding RNA. The conducted study focuses on the miR30 family, with particular attention to miR-30e, which, from previous studies, has been observed to be under expressed in mice cells affected by the SOD1 G93A mutation. Through in silico techniques, it has been understood that a probable interactor of this RNA is another non-coding RNA, specifically the lncRNA Gas5, which is overexpressed in the same murine cells. Therefore, the effects of miR30e were investigated in immortalized mouse cells (C2C12), observing that the resulting myotubes showed a higher fusion index and thickness. This effect was observed both directly and indirectly through co-culture, indicating that factors inducing greater fusion are also released through paracrine communication. MiR30e, therefore, could be considered and investigated as a potential candidate for gene therapy aimed at slowing down the symptoms of ALS.