Seminars Archive

Towards a molecular understanding of neurodegenerative diseases

Abstract
An increasing family of neurodegenerative disorders such as Alzheimer\'s, Parkinson\'s and Huntington\'s diseases, prion encephalopathies, and cystic fibrosis has recently been associated with aggregation of misfolded polypeptide chains which are toxic to the cell [1], eventually leading to neuronal death. All members of this family of diseases are linked to a mechanism of protein misfolding, therefore suggesting the tremendous importance of understanding the structure and functions of the proteins implicated, both in their healthy and in their pathological forms. Yet, relatively little is still known about the mechanisms of aggregation and the events which trigger it, mostly because of the specific difficulties that such studies imply. In my talk, I shall review the field of aggregation and illustrate in detail a specific example, taken from my own research experience, of how structural studies may provide a powerful tool to approach misfolding diseases. 1. Temussi, P.A., Masino L.and Pastore A. From Alzheimer to Huntington: why is a structural understanding so difficult? EMBO J. 22, 355-361 (2003)