Using human genomics to identify drug targets for progressive MS

MS is a progressive disease that damages the central nervous system (CNS), causing inflammation and loss of protective myelin around nerve cells. While treatments exist to manage early MS, options are very limited for people with progressive MS. Associate Professor Justin Rubio’s research aims to tackle a major hurdle that has slowed the development of therapies that could protect or repair the brain in progressive MS.

Associate Professor Rubio’s team has contributed significantly to understanding MS at the genetic level. Recently, they discovered that brain cells in MS lesions accumulate genetic mutations much faster than normal. This discovery suggests that certain genetic changes in the brain may influence how MS progresses. Their recent genome-wide association study (GWAS) on MS also indicates that the brain’s ability to adapt, or its resilience, plays an important role in MS progression.

Building on these insights, Associate Professor Rubio and his team will use advanced methods to analyse genetic mutations, gene expression, and regulation in the brain. By combining these data with their GWAS findings, they aim to identify genes and pathways involved in MS that could serve as targets for new treatments. The team will use computational tools to find these potential targets, drawing on public data as well as their expertise in MS research, from laboratory work to clinical application.

This project could pave the way for new therapies that protect brain cells and slow MS progression, offering hope for people with progressive MS.