- Researchers from the University of Montpellier, supported by the AFM-Telethon, have shown that muscle cell damage in oculopharyngeal muscular dystrophy (OPMD) is mediated by overactivation of the proteasome leading to muscle protein degradation, rather than by the accumulation of mutated PABPN1 proteins.
- They then provided proof of concept in Drosophila larvae that proteasome inhibitors, such as MG132, can ameliorate muscle damage in models of PCOD.
- These drugs, used as anti-cancer drugs, offer new hope for the future treatment of PCOD.
Activation of the ubiquitin-proteasome system contributes to oculopharyngeal muscular dystrophy through muscle atrophy. Ribot C et al. PLoS Genet. 2022 Jan
