Journal
JOURNAL OF MEDICINAL CHEMISTRY
Volume 53, Issue 3, Pages 1407-1411Publisher
AMER CHEMICAL SOC
DOI: 10.1021/jm9013345
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Funding
- INSERM
- FRM
- University of Paris Descartes
- Association Francaise contre les Myopathies, Paris, France
- Canadian Institutes of Health Research
- Muscular Dystrophy Association
- Foundation of Greater Philadelphia
- FIRB
- RETI FIRB
- Fondazione Roma
- AIRC
- EU [HEALTH-174-2007200767, HEALTH-F2-2007-200620, HEALTH-F4-2009-221952]
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In oculopharyngeal muscular dystrophy (OPMD), a disease caused by polyalanine expansion in the nuclear protein PABPN1, the genetic inhibition or sirtuins and treatment with sirtuin inhibitors protect from mutant PABPN1 toxicity in transgenic nematodes. Here, we tested the SIRT1/2 inhibitors 1-12, bearing different degrees of inhibition, for protection against mutant PABPN1 toxicity in Caenorhabditis elegans. Compounds 2, 4, and 11 were the most efficient, revealing a potential therapeutic application for muscle cell protection in OPMD.
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