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Huntington's disease (HD) is caused by a dominant mutation in HTT, the HD gene. This discovery opens possibilities for treatment based on silencing of the disease-causing allele or with compounds that reduce the production of disease-causing mRNA and/or protein. Although additional developments are needed related to the delivery of gene silencing and discovery and development of drugs that reduce disease-causing gene products, these treatments are predicted to be effective since they act by reducing the source of toxicity. The identification of therapies that act by blocking toxicity is conceptually more complicated, as this requires an accurate understanding of the cellular location and the specific molecular dysfunctions that cause the phenotypes of HD, which is not yet available. Though challenges remain, significant progress has been made. Effective disease-modifying treatments will soon be tested and may lead to disease-altering therapies.

Huntington's disease: progress toward effective disease-modifying treatments and a cure