In-vitro and in-vivo animal and human-based pre-clinical study on a multiple-miRNA-targeted therapy designed by chemical reaction network computational analysis andexploited by nanovector technology

The causes of amyotrophic lateral sclerosis (ALS) are not well understood. Recent studies have shown that circulating small biological fragments of RNA that can influence gene activity, called microRNAs (miRNAs) could be used as markers of disease and therapeutic targets. The levels of certain miRNAs are different in ALS patients, and small vesicles that shuttle miRNAs between cells play a key role in their transport and transfer. This suggests that there may be a link between these abnormal miRNAs and possible ways to treat the disease. Although there have been some recent advances, there is still a need for new therapies. Our research proposes a multi-faceted approach through informatic analyses applied to biological systems and the development of biocompatible nano vectors for targeted delivery of synthetic molecules that mimic miRNAs. The aims include finding out which miRNAs are important for ALS, checking their effects using human- and mouse-derived cells, and doing in vivo experiments to test if these innovative nano vectors can help treat ALS mice. This project aligns with the MNDA's strategy to find new ways to cure ALS by combining advanced bioinformatic methods and new ways to deliver drugs, understand how miRNAs work in the disease, and create new ways to treat ALS and other similar pathologies of the nervous system.