"The successful restoration of visual function with recombinant adeno-associated virus-mediated gene replacement therapy has ushered in a new era of retinal therapeutics," said Andr-s M. Kom-romy, assistant professor of ophthalmology at the Penn School of Veterinary Medicine and lead author of the study.

Many vision-impairing disorders in humans result from genetic defects, and, to date, mutations have been identified in ~150 genes out of ~200 mapped retinal disease loci. This wealth of genetic information has provided fundamental understanding of the multiple and specialized roles played by photoreceptors and the retinal pigment epithelium in the visual process and how mutations in these genes result in disease. Together with the development of gene-transfer technologies, it is now possible to realistically consider the use of gene therapy to treat these previously untreatable disorders.

Source: University of Pennsylvania