title: Retinal Gene Therapy: Current Progress and Future Prospects.
abstract: Clinical trials treating inherited retinal dystrophy caused byRPE65mutations 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.
authors.affiliation: Center for Neuroscience, Robert C. Byrd Health Sciences Center, West Virginia University, Morgantown, West Virginia, 26505, USA.
authors.forename: Cristy A
authors.initials: CA
authors.lastname: Ku
authors.affiliation: Casey Eye Institute, Oregon Health & Science University, Portland, OR, 97239, USA.
authors.forename: Mark E
authors.initials: ME
authors.lastname: Pennesi
keywords: RPE65; adeno-associated virus; equine infectious anemia virus; gene therapy; inherited retinal dystrophies