Researchers identify a cellular pathway that clears toxic TDP-43
Original source
Rsp5/NEDD4 and ESCRT regulate TDP-43 toxicity and turnover via an endolysosomal clearance mechanism. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human, Animal
Related topics
A study found that Rsp5/NEDD4 and ESCRT proteins help remove cytoplasmic TDP-43 through the endolysosomal system. Disrupting these proteins increased TDP-43 aggregation, stability and toxicity in experimental models.
Why this matters
TDP-43 mislocalisation and aggregation is reported as a hallmark of more than 97% of ALS cases. The findings point to a possible therapeutic strategy for improving TDP-43 clearance, but they do not yet change treatment for people with ALS.
Limitations and context
This was laboratory research that included an unbiased screen in yeast and experiments in ALS models. The study did not show that targeting this pathway is safe or effective in people, so clinical benefit remains unestablished.
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