title: What Is Next for Retinal Gene Therapy?
abstract: The field of gene therapy for retinal blinding disorders is experiencing incredible momentum, justified by hopeful results in early stage clinical trials for inherited retinal degenerations. The premise of the use of the gene as a drug has come a long way, and may have found its niche in the treatment of retinal disease. Indeed, with only limited treatment options available for retinal indications, gene therapy has been proven feasible, safe, and effective and may lead to durable effects following a single injection. Here, we aim at putting into context the promise and potential, the technical, clinical, and economic boundaries limiting its application and development, and speculate on a future in which gene therapy is an integral component of ophthalmic clinical care.
authors: Ocular Genomics Institute, Grousbeck Gene Therapy Center, Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston, Massachusetts 02114.
authors: Luk H
authors: LH
authors: Vandenberghe
mesh_terms: D002986:Clinical Trials as Topic; D005544:Forecasting; D015316:Genetic Therapy / Q000191:economics / Q000379:methods / Q000639:trends*; D006801:Humans; D012162:Retinal Degeneration / Q000191:economics / Q000235:genetics / Q000628:therapy; D012164:Retinal Diseases / Q000191:economics / Q000235:genetics / Q000628:therapy*; D019076:Transgenes / Q000235:genetics; D016896:Treatment Outcome