The evolutionary conserved circHTT(2,3,4,5,6) as a new disease modifier in Huntington’s Disease pathology

Still no treatment is available to halt Huntington’s disease (HD). However, a world-wide effort is ongoing to identify strategies to lower the mutant huntingtin protein and to modulate crucial players, ‘modifiers’ of the disease.
Our project focuses on a novel circular RNA molecule – circHTT – produced from the same region undergoing mutation in HD. CircHTT, recently discovered by our group, is very stable because of its circular structure, found at high levels in the brain and strongly increased in HD conditions.
Our goal is to illustrate how circHTT functions and what its modulation (i.e. down regulation of its expression) might cause to cell and neurons. These observations will possibly identify a new tool to control protein production and, eventually, modifying cellular features associated with the disease.