2016 ALS Association Investigator-Initiated Starter Grant
Dr. Kausiki Datta and Dr. Andrew Berglund have been awarded the 2016 Investigator-Initiated Starter Grant by the ALS Association, to screen for novel compounds to treat Amyotrophic Lateral Sclerosis (ALS).
In this project, Dr. Datta, in collaboration with Dr. Berglund and Dr. Laura Ranum, will optimize a high-throughput screening assay for identifying small molecules that reduce or eliminate the production of G4C2/G2C4 hexanucleotide RNA repeat expansions linked to the molecular pathogenesis of ALS. Targeting transcription to downregulate the production of repeat RNAs will have significant therapeutic potential as this should correct many of the downstream toxic effects, including splicing defects and production of toxic RAN proteins. By utilizing a high-throughput screening approach, the group hopes to identify novel compounds that will yield insights into developing potential therapeutic strategies for ALS.