A transcriptional report card from the peripheral blood: Can it measure disease progression in Huntington's disease?

摘要

As the underlying molecular basis for genetically based neurodegenerativedisorders such as Huntington's disease (HD) has been revealed, these diseases now present critical new challenges to workers in the field. While the development of effective interventions to limit or halt the inexorable progression of the disease process has become a central research focus, the ability to do accurate and efficient clinical trials has also emerged as a key rate-limiting step in the development of an effective clinical intervention.Current functionally based measures of disease status and progression have limited sensitivity. A more directly biologically based measure of disease status and progression might significantly increase the power and reduce the time required for clinical trials for neurodegenerative disease. In a recent study, Borovecki et al took a novel approach to this problem, by focusing their attention far from the primary site of pathology in HD, the brain, and instead looked to the peripheral blood, using DNA microarrays to identify a set of mRNAs that appeared useful as biomarkers to reflect disease status and progression for HD.