Targeting autophagy
Dr Christopher Sibley, The University of Edinburgh
Advancing Treatments Award
Accelerate new treatments
April 2025 - April 2028
£298,619.74
Summary
TDP-43 is a protein that plays an important role in the assembly line that converts information stored in our DNA into molecules called RNA, which are used as the template for making the proteins within our cells. While TDP-43 is known to be dysregulated in about 97% of people living with MND, we still don’t have an effective way to target it therapeutically.
Dr Sibley and his team previously identified that TDP-43 causes changes in the RNA encoding a protein called ATG4B involved in autophagy, the process of cellular recycling. Autophagy is known to be dysregulated in MND. Changes in the ATG4B gene result in lower ATG4B protein expression and this in turn increases the aggregation of TDP-43 into toxic clumps in motor neurons.
In this project, the team will explore the development of a gene therapy targeting ATG4B RNA, with the hope to validate it as a target for future MND treatments.
Why this research is important
This project will help us understand whether autophagy could be a good target for future MND treatments, opening up the door for a potential new treatment avenue.
To learn more about TDP-43 take a look at our TDP-43 infographic.
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