Suppression of Somatic Expansion As a Novel Therapeutic Approach for Huntington Disease and Other Repeat Expansion Disorders

Huntington disease (HD) is one of a growing number rare genetic diseases characterized by the inheritance an increased short tandem repeats within affected gene. Many these repeat expansion disorders (REDs) affect brain. Although mutant allele necessary first step for manifestation, second further inherited expanded repeats, particularly in neurons brain, also appears to play critical role toward manifestation. This dynamic process, called somatic expansion, modulated genes involved repair DNA that operate upstream specific gene associated with each individual disease. As has been described multiple REDs, regulating are attractive therapeutic targets, since REDs could potentially be treated same drug.