New agents inactivate RNA from mutated gene in ALS and FTD

08/14/2014 | Medical News Today

Two of the three novel small-molecule agents designed by researchers with the Scripps Research Institute and the Mayo Clinic reduced the burden of c9RAN, a toxic protein associated with amyotrophic lateral sclerosis and frontotemporal dementia, according to a study in the journal Neuron. The toxic proteins are encoded in a mutated form of the C90RF72 gene, present in both disorders. The molecules work by inhibiting the encoded RNA's ability to interact with other vital proteins.

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