Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Parkinson's disease (PD)

Parkin (PARK2); ubiquitin specific peptidase 30 (USP30)

Cell culture and fly studies suggest inhibiting USP30 could help treat PD. In a mitochondrial degradation assay using cultured human dopaminergic neurons, USP30 inhibited PARK2-mediated mitophagy, a process whereby damaged mitochondria get degraded that is impaired in PD. In the cultured human dopaminergic neurons, USP30-targeting siRNA rescued PD-associated impairments in mitophagy, whereas control siRNA did not. In a fruit fly model of PD, usp30 knockdown improved motor function and increased both mitophagy and survival compared with no alteration. Next steps include evaluating USP30 inhibition in mammalian models and identifying pharmacodynamic markers of USP30 inhibition.

SciBX 7(25); doi:10.1038/scibx.2014.745
Published online June 26, 2014

Patent and licensing status undisclosed

Bingol, B. et al. Nature; published online June 4, 2014;
doi:10.1038/nature13418
Contact: Baris Bingol, Genentech Inc., South San Francisco, Calif.
e-mail:

bingol.baris@gene.com