Huntington disease (HD) is caused by a cytosine-adenine-guanine trinucleotide repeat
expansion in the huntingtin gene, HTT, that results in expression of variant (mutant)
huntingtin protein (HTT). Therapeutic strategies that reduce HTT levels are currently being
pursued to slow or stop disease progression in people with HD. These approaches are
supported by robust preclinical data indicating that reducing variant huntingtin protein is
associated with decreased HD pathology. However, the risk-benefit profile of reducing either …