Using human genomics to identify drug targets for progressive MS

Associate Professor Justin Rubio

THE FLOREY INSTITUTE OF NEUROSCIENCE AND MENTAL HEALTH, VIC

| A cure via repair and regeneration | Epidemiology | Genetics | Fellowship | 2024 | Investigator Led Research |
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Summary

Multiple sclerosis (MS) is a common cause of neurological disability in young adults, affecting over 33,000 people in Australia and over 2.8 million people globally. There are treatments for relapsing forms of MS, but these are mostly ineffective in treating people living with progressive MS (PMS).

Associate Professor Justin Rubio and his team will integrate several types of genomic data, from single cells and human populations, to uncover genes that may be involved in progressive MS and how they fit into the MS puzzle. They will then conduct gene targeting in human stem cells using innovative laboratory techniques to explore the function of these genes in models of MS.

From this research, Associate Professor Rubio and his team hope to discover potential drug targets for further investigation. It is expected that this will build the case for translation into clinical trials where newly developed drugs are tested in people living with progressive MS in the hope of slowing or preventing progression.

Updated: 22 January, 2024

Stages of the research process

Fundamental laboratory
Research

Laboratory research that investigates scientific theories behind the possible causes, disease progression, ways to diagnose and better treat MS.

Lab to clinic timeline: 10+ years
Translational
Research

Research that builds on fundamental scientific research to develop new therapies, medical procedures or diagnostics and advances it closer to the clinic.

Lab to clinic timeline: 5+ years
Clinical Studies
and Clinical Trials

Clinical research is the culmination of fundamental and translational research turning those research discoveries into treatments and interventions for people with MS.

Lab to clinic timeline: 1-5 years

Total Funding

  • $650,000

Duration

  • 5 years – starting 2024

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Using human genomics to identify drug targets for progressive MS