Final Report Summary - MECHAGGRENAMICS (Mechanisms of cell dysfunction by aggregation dynamics of polyQ-containing proteins)
In this proposal we have generate new in vivo models of HD to find molecules that modulate protein and RNA toxicity and disease progression. We also have tested several compound that reduce aggregation patterns, and also induce neuronal protection in contexes of polyQ toxicity.
We have found seeveral genes involved in protein toxicity dynamics, and also in genes that are involved in the toxicity produced by the nascent RNA containing expanded CAG triplets.
We are currently testing drugs against HD in a mice model of HD, the so-called zQ175. Some of these compounds will be considered for future clinical trials.
More information at the website of our lab: www.biomcg-wormlab.org