Developing novel frontotemporal dementia and amyotrophic lateral sclerosis therapeutics to rescue TDP-43 mistranslocation
Disease area:
MND and FTD
Research Project Description
The trans-activation response DNA-binding protein (TDP-43) is a DNA/RNA-binding protein located in the nucleus involved in regulating alternative splicing processes and transcriptional activities of RNA. In amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), a common key pathological hallmark is the abnormal mislocalization and aggregation of TDP-43.
In vivo studies have shown that the selective reduction of mislocalized TDP-43 from the cytoplasm is linked to reduced toxicity. Since there are currently no treatments reverse ALS and FTD, TDP-43 represents a promising target to develop novel therapeutic options.
Using western blot and binding assays on purified recombinant TDP-43. The aim is to understand the binding and function of novel compounds on TDP-43.