this post was submitted on 17 Nov 2023
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This study reveals that inhibiting a specific enzyme, USP30, in a mouse model can protect dopamine-producing neurons. This finding halts the disease’s progression and opens the door to new therapeutic possibilities for the 10 million people affected by Parkinson’s worldwide.

DOI: 10.1038/s41467-023-42876-1

https://www.nature.com/articles/s41467-023-42876-1

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[–] [email protected] 6 points 11 months ago* (last edited 11 months ago)

"In conclusion, we used a strategy for upregulating mitophagy by Usp30 deletion, with similar results obtained with a pharmacological USP30 inhibitor. These strategies to reduce USP30 lead to enhanced mitophagy and potent protection against αSyn toxicity. This work validates inhibition of USP30 as a promising strategy for further testing for potential disease-modifying effects in PD."

Mitophagy - clearing out old stuff from cells. Clears out dysfunctional mitochondria from cells.

aSyn toxicity - alpha synuclein - a protein found in the body, esp. in the brain. [We think it helps with some neruo-regulation, and DNA repair. We think these two go together. ] In people with diseases like Alzheimers an Parkinsons there appears a 'buildup' of αlpha-synuclein in places and a corresponding degeneration of other areas.

This study tried to crank up the clearing out of old mitochondria in Mice first by modifying their genes to removing (knocking out) the ability to make USP30 (an enzyme). They also cooked up a molecule that could just block USP30 (it gets there first and sits in the way).

Both methods worked to protect mice when they tried to give them alpha synuclein problems. Of course there's lots of other questions about what else is affected, but this has potential to be fix this one part of the problem.