463. Safety and Biodistribution Study of rAAV2tYF-CB-hRS1 in RS1-deficient Mice

Intravitreal administration of rAAV2tYF-CB-hRS1 in RS1-deficient mice was well tolerated with minimal to slight ocular inflammatory cells detected by histopathology. RS1 expression demonstrated by immunohistochemistry was associated with decreased severity of splitting/disorganization of the inner nuclear layer of the retina at the higher dose level. These results support the use of rAAV2tYF-CB-hRS1 in clinical studies in patients with XLRS.