Causes of death among patients with AAV were most often attributed to infection, though discrepancies were found between chart review and death certificate data. Significant discrepancies exist ...

Secondly, any pre-existing T cell immunity to AAV vectors can result in acute cytotoxic T cell responses that can substantially reduce the effectiveness of a therapy. Finally, acute toxicities ...

thus potentially also increasing the therapeutic potency of the new therapies. "To achieve this goal, we studied how AAV vectors influence the rate of gene editing and have used that knowledge to ...

However, system upgrades to the leading gene delivery vehicle offer hope that effective therapies will soon be within reach. There is a growing list of approved AAV-based gene therapies and hundreds ...