Defective metabolism of sialo-conjugates in Huntington’s disease is a druggable target for the development of new effective therapeutic strategies

Huntington disease (HD) is rare fatal hereditary brain disorder with no effective cure available. Our past studies along with our preliminary data demonstrate that the disease is characterized a significant alteration in the biology of molecules that are linked to sialic acid, a special sugar very important for the brain functions. 
The main purpose of this proposal is demonstrate that  such a defect  represents an important target for developing new and effective therapeutic options for the disease.
For the achievement of our objectives we will use multiple study models, including cells from human HD patients, that will be analyzed by multiple multidisciplinary approaches.
We believe that our proposal has the potential to provide some new insight into the pathogenic mechanisms underlying HD and, to make available new valuable therapeutic options for its treatment in the future.