Gene Therapy Rescues Cone Structure and Function in the 3-Month-Old<i>rd12</i>Mouse: A Model for Midcourse RPE65 Leber Congenital Amaurosis

Abstract

The results support and extend those of the previous study that gene therapy can stop early cone degeneration, and, more important, they provide proof that delayed treatment can restore the function and morphology of the remaining cones. These results have important implications for the ongoing LCA2 clinical trials.