Peripheral biomarkers in prodromal and Huntington's disease progression: predictive potential and drug-screening approach for treatment repurposing
In HD field identifying biomarkers (in addition to the number of GAG repeats) may predict age of onset and disease progression. Recently easily accessible predictors, coming from peripheral leucocytes and plasma, are receiving attention.
We got informative preliminary results on DNA damage response (DDR) and the telomeres shortening in peripheral leucocytes, along with circulating micro-RNAs and small nucleolar RNAs. All these biomarkers will longitudinally (3 years) be studied in pre-manifests, HD patients, and matched controls, and will be integrated through a biostatistics approach to measure their predictive potential. Moreover, some evidences on DDR in peripheral leucocytes from pre-manifests (it increases over time and collapsed, although at higher than normal levels, once the symptoms appeared; it is absent in healthy subjects and it is potentially reversible) supports its use as quick and handy drug-screening approach to explore treatment repurposing before disease onset.