SEMINAR: In silico reconstruction of ancestral adeno-associated viruses for retinal gene therapy

Adeno-associated virus vectors (AAV) are a safe and effective tool for gene therapy, particularly for the treatment of inherited blindness. However AAVs are endemic in the human population and its efficiency is subjected to circulating neutralizing antibodies which significantly limit broader applications of this promising approach. Under the hypothesis that ancestral AAV particles were more resistant to contemporary pre-existing immunity we have used maximum likelihood methods to reconstruct a probabilistic space around an ancestral node of the AAV phylogeny. This talk will focus on characterizing the retinal tropism of these viruses and their role in providing an alternative system for future use in human gene therapy approaches.