I039 Genetic engineering to the rescue of HD

<h3></h3> The combined use of gene transfer and editing technologies has pushed the boundaries of precise genome modification and fostered the development of promising strategies for the treatment of genetic diseases, including Huntington’s disease. In this context, I will illustrate: a) the combined use of adeno-associated vectors (AAV) and the KamiCas9 self-inactivating gene editing to target the corticostriatal neuronal network affected in HD, b) the use of 3D and 2D semi-automated workflows to predict the degree of huntingtin (HTT) gene inactivation in multiple brain regions, and c) finally the long-term consequences of wild-type HTT inactivation in striatal and projection neurons of adult mice.