PROJECT 11:

Host: Siena Biotech

 

 

PI:
Andrea Caricasole (at associated partner Promidis)

Fellow:
Lara Petricca

Amongst the neurodegenerative diseases, PolyQ-diseases share common features and are characterized by the presence of an expanded polyglutamine tract in the mutant proteins, altering their biological properties.

We are investigating Hungtington’s Disease (HD) as a model for PolyQ-diseases.

HD is a rare, autosomal dominantly inherited neurodegenerative brain disorder with a high “unmet medical need” as its patho-physiology is still largely unknown and no efficacious disease- modifying therapy exists.

Our project aims at investigating posttranslational modifications of the full length protein huntingtin, normal and mutant. Our goal is to set up tools to measure levels of huntingtin and to identify huntingtin-modifying enzymes capable of altering huntingtin protein levels as therapeutic targets for HD.