Small molecule drug discovery for Huntington's Disease.

摘要: Huntington's Disease (HD) is a rare neurodegenerative disease caused by mutation of the huntingtin gene that results in protein with an expanded stretch glutamine repeats (polyQ). Knowledge validated targets its infancy, and thus, traditional target-based drug discovery strategies are limited use. Alternative approaches needed, early attempts were aimed at identifying molecules inhibited aggregation polyQ fragments. More recently, phenotypic assays used to find able reverse some pathogenic mechanisms HD. Such have impact on configuration screening cascades for effective translation candidates toward clinical trials.