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

Abstract excerpt

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...

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