Article
A yeast-based assay identifies drugs active against human mitochondrial disorders.
Proceedings of the National Academy of Sciences of the United States of America - 19 Jul 2011
Couplan Elodie, Aiyar Raeka S, Kucharczyk Roza, Kabala Anna, Ezkurdia Nahia, Gagneur Julien, St Onge Robert P, Salin Bénédicte, Soubigou Flavie, Le Cann Marie, Steinmetz Lars M, di Rago Jean-Paul, Blondel Marc
Abstract excerpt
Due to the lack of relevant animal models, development of effective treatments for human mitochondrial diseases has been limited. Here we establish a rapid, yeast-based assay to screen for drugs active against human inherited mitochondrial diseases affecting ATP synthase, in particular NARP (neuropathy, ataxia, and retinitis pigmentosa) syndrome. This method is based on the conservation of mitochondrial function...
Topics
- Cell Line
- Chlorhexidine
- Drug Discovery
- Drug Evaluation, Preclinical
- Gene Expression Profiling
- Humans
- Mitochondrial Myopathies
- Mitochondrial Proton-Translocating ATPases
- Mutagenesis, Site-Directed
- Mutation
- Oleic Acid
- Retinitis Pigmentosa