University of Pennsylvania Medical Center preclinical data

Researchers at the University of Pennsylvania Medical Center and colleagues reported data from a study in 6 dogs with congenital blindness and 4 healthy monkeys showing that AAV2-hRPE65v2 administered to the left eye 14 days after treating the right eye was safe with no evidence of unwanted immune reactions following the previous injection. Specifically, no animals developed antibodies against the transgene product, but all developed neutralizing antibodies against the adeno-associated virus (AAV) capsid in sera and intraocular fluid. Cell-mediated immune responses were benign, with only 1 animal developing a persistent T cell immune response to the capsid. ...