title: Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.
abstract: Over the past 20 years, there has been tremendous progress in retinal gene therapy. The safety and efficacy results in one early-onset severe blinding disease may lead to the first gene therapy drug approval in the United States. Here, we review how far the field has come over the past two decades and speculate on the directions that the field will take in the future.
authors.affiliation: Center for Advanced Retinal and Ocular Therapeutics (CAROT) and F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA. Electronic address: jebennet@mail.med.upenn.edu.
authors.forename: Jean
authors.initials: J
authors.lastname: Bennett
mesh_terms: D000260:Adenoviruses, Human / Q000235:genetics* / Q000378:metabolism; D000818:Animals; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D017277:Drug Approval; D005136:Eye Proteins / Q000235:genetics* / Q000378:metabolism; D015870:Gene Expression; D015316:Genetic Therapy / Q000379:methods / Q000639:trends*; D005822:Genetic Vectors / Q000008:administration & dosage* / Q000737:chemistry / Q000378:metabolism; D006801:Humans; D056965:Injections, Intraocular; D009154:Mutation; D012160:Retina / Q000378:metabolism / Q000473:pathology; D012164:Retinal Diseases / Q000235:genetics / Q000378:metabolism / Q000473:pathology / Q000628:therapy*
chemical_list: D005136:Eye Proteins
keywords: clinical trials; gene therapy; retina