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Huntington's disease (HD) is a rare neurodegenerative disease impacting approximately 2.7 individuals out of 100,000 worldwide. However, it presents an appealing focus for nucleic acid-targeting therapies. This is due to its origin in an expanded DNA repeat, which, when transcribed, produces harmful repeat RNA. Thus, we hypothesized that a compound that binds to the disease-causing DNA and inhibits transcription to prevent the formation of toxic RNA could be promising as a HD therapeutic. By preventing the formation of toxic RNA, the downstream translation that forms homopolymeric proteins could be knocked down, potentially diminishing protein aggregates that often cause HD symptoms and disease progression. This investigation focuses on a specific compound functioning as a groove binder. In this study we tested the compound of interest in Hela Cells transfected with the pEGFP-Q74 plasmid. A fluorescence microscope was used to determine if the amount of protein aggregates increased or decreased in the presence of our compound in a normal length repeat as compared to the disease length. Thus, this work contributes to understanding the efficacy of this compound as a treatment for HD.
Berko et al. (Fri,) studied this question.