Clinical trials treating inherited retinal dystrophy caused by RPE65 mutations 
had put retinal gene therapy at the forefront of gene therapy. Both successes 
and limitations in these clinical trials have fueled developments in gene 
vectors, which continue to further advance the field. These novel gene vectors 
aim to more safely and efficiently transduce retinal cells, expand the gene 
packaging capacity of AAV, and utilize new strategies to correct the varying 
mechanisms of dysfunction found with inherited retinal dystrophies. With recent 
clinical trials and numerous pre-clinical studies utilizing these novel vectors, 
the future of ocular gene therapy continues to hold vast potential.