Obiettivo The description of the initial molecular events in Huntington's disease is crucial to design therapeutic intervention for the cure of this devastating disorder. In this proposal, I plan to identify the early HD-trigger mechanism, aiming for the development of rational therapeutics. Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder due to an expanded, unstable CAG repeated sequence that elongates a segment of glutamine residues in the HD gene's protein product, huntingtin.The disease is characterized by progressively worsening chorea, psychiatric impairment and cognitive decline. The most striking pathological manifestation of HD is a specific and gradual loss of neurons, predominantly in the caudate nucleus and putamen of the brain. I propose, via a combinatorial screening, to isolate small peptides that specifically bind mutant huntingtin and that are able to reverse in vivo pathogenic phenotypes by displacing crucial protein-protein interactions.An aptamer with such characteristics will represent a very exciting lead to design small drugs for therapeutic intervention. I anticipate that this study will provide a better understanding of the first molecular events of the disease, providing suitable targets for therapeutic intervention. Campo scientifico natural sciencesbiological sciencesneurobiologynatural sciencesbiological sciencesbiochemistrybiomoleculesproteinsproteomicsmedical and health sciencesmedical biotechnologycells technologiesstem cellsmedical and health sciencesclinical medicinecardiologycardiovascular diseases Programma(i) FP6-MOBILITY - Human resources and Mobility in the specific programme for research, technological development and demonstration "Structuring the European Research Area" under the Sixth Framework Programme 2002-2006 Argomento(i) MOBILITY-4.2 - Marie Curie International Reintegration Grants (IRG) Invito a presentare proposte FP6-2002-MOBILITY-12 Vedi altri progetti per questo bando Meccanismo di finanziamento IRG - Marie Curie actions-International re-integration grants Coordinatore INTERNATIONAL SCHOOL FOR ADVANCED STUDIES Contributo UE Nessun dato Indirizzo Via Beirut 2-4 TRIESTE Italia Mostra sulla mappa Collegamenti Sito web Opens in new window Costo totale Nessun dato
