Ben Deverman, Caltech

Recombinant adeno-associated viruses (rAAVs) have become essential tools for in vivo gene delivery. In this talk, I will describe how we have used a novel cell-type-specific in vivo selection method to engineer AAV-PHP.eB and AAV-PHP.S, vectors capable of efficient gene transfer throughout the central and peripheral nervous systems, respectively. In addition, I will briefly discuss how we are engineering AAVs for greater cell-type specificity, and how we are evaluating these new AAVs as vehicles for the treatment of neurodegenerative diseases.