Amyotrophic Lateral Sclerosis (ALS) can result from many different mutations in the same gene, making mutation-specific treatments challenging. This project focuses on using antisense oligonucleotides (ASOs) to selectively target mutant RNA from the VCP gene, reducing production of the harmful protein while preserving the normal version. To overcome the major limitation of ASOs, which is their inability to cross the blood-brain barrier, the team is developing a non-invasive carrier system to deliver ASOs directly to the brain. This work aims to advance ALS treatment and improve ASO delivery for other neurodegenerative diseases.
