We previously reported that the systemic administration of mouse hematopoietic bone marrow-derived progenitor cells (HSPCs), defined by the surface markers cKit+Sca-1+, when infected ex vivo with a lentiviral vector expressing the RPE-specific gene RPE65 (LV-RPE65), demonstrate rapid recruitment to the injured retina and improve visual function by restoring a functional RPE layer in both acute and chronic murine models of retinal degeneration [1,2,3]. Here, RPE65 is linked to retinal degeneration.