Abstract 2131 Utilizing Potential Therapeutic Compounds to Inhibit Transcription Contributing to Huntington's Disease

Huntington's disease (HD) is a rare genetically inherited disorder that affects about 1 in 10,000 people worldwide. In addition to the potential long-term benefit to patients, HD is interesting to study due to the potential to experiment with targeting the disease at the nucleic acid level. Because symptoms of HD are caused by protein aggregates, the hypothesis of this experiment is that binding nucleic acids can prevent the production of the toxic protein aggregates by preventing transcription and/or translation. In this study, a transcription inhibition assay was used to investigate the efficacy of three compounds as potential nucleic acid-targeting agents. The compounds were tested against a disease-relevant sequence and a random sequence, and the lead compound showed selective inhibition at low micromolar concentrations. These results support the potential of this compound as a therapeutic agent for HD and opens avenues for further testing. This work was supported by Mount St. Mary's startup funds for Dr. Krueger.