1035. Short G-Rich Oligonucleotides as a Therapeutic for Huntingtonss Disease

Abstract

Huntington's Disease (HD) is an inherited autosomal dominant genetic disorder in which neuronal tissue degenerates. The pathogenesis of the disease appears to center on the development of protein aggregates that arise initially from the misfolding of the mutant HD protein. Mutant huntingtin (Htt) is produced by HD genes that contain an increased number of glutamine codons and this expansion leads to the production of a protein that misfolds. Recentstudies suggest that mutant Htt can nucleate protein aggregation and interfere with a multitude of normal cellular functions.