AAV Biology
Despite intense efforts of numerous laboratories many aspects of AAV biology remain unexplored or are controversial. Our laboratory focuses in particular on the elucidation of the endocytic pathway(s) of AAV and the intracellular trafficking paths that lead to a successful transduction of a cell. (Read More)
Creation of AAV Variants with Improved Properties
The various serotypes of AAV display broad but distinct tissue/cell tropism. Nonetheless, some cells remain refractory to AAV transduction, and all presently known AAVs infect more than one cell type in the body. Moreover, a significant segment of the population harbors pre-existing neutralizing antibodies against AAVs, which precludes them from being included into clinical trials or the clinical use of approved AAV gene therapies. In this area of our research, we aim to address these challenges. (Read More)