MFN2 Stabilization via Calpain-Resistant Gene Therapy in ALS

This concept paper was developed independently by a non-scientist with no formal training, based on personal research into ALS beginning in 2022. It explores a possible gene therapy approach for ALS that aims to protect a protein called MFN2, which helps keep mitochondria healthy and supports the connection between nerves and muscles. The idea is to prevent this protein from being broken down by an enzyme called calpain, which becomes overactive in ALS. The modified MFN2 would be delivered using a type of viral vector known for reaching motor neurons effectively. This is a non-commercial effort, created out of personal interest, with the hope that the idea might be useful to researchers working in this field.