AAV Vector Development

The Sena-Esteves laboratory aims to improve current limitations to AAV-based gene therapy for CNS diseases in two ways: 1) manipulation of the blood brain barrier (BBB) for increased accessibility of vectors to the brain after systemic injection, and 2) screening for novel AAV capsids with desired properties. These properties include the ability to cross the BBB following systemic infusions, high gene transfer efficiency to the CNS, particularly neurons, and evasion of neutralizing antibodies prevalent in the patient population. Novel directed evolution library approaches are employed to identify capsids that meet our criteria.