Gene Therapy and Stem Cell Transplantation in Retinal Disease: The New Frontier.

ABSTRACT: Gene and cell therapies have the potential to prevent, halt, or reverse diseases of the retina in patients with currently incurable blinding conditions. Over the past 2 decades, major advances in our understanding of the pathobiologic basis of retinal diseases, coupled with growth of gene transfer and cell transplantation biotechnologies, have created optimism that previously blinding retinal conditions may be treatable. It is now possible to deliver cloned genes safely and stably to specific retinal cell types in humans. Preliminary results testing gene augmentation strategies in human recessive diseases suggest promising safety and efficacy profiles, including improved visual function outcomes over extended periods. Additional gene-based strategies under development include approaches to

SUBMITTER: MacLaren RE 

PROVIDER: S-EPMC5545086 | biostudies-literature | 2016 Oct

REPOSITORIES: biostudies-literature