title: Advances in Gene Therapy for Diseases of the Eye.
abstract: Over the last few years, huge progress has been made with regard to the understanding of molecular mechanisms underlying the pathogenesis of neurodegenerative diseases of the eye. Such knowledge has led to the development of gene therapy approaches to treat these devastating disorders. Challenges regarding the efficacy and efficiency of therapeutic gene delivery have driven the development of novel therapeutic approaches, which continue to evolve the field of ocular gene therapy. In this review article, we will discuss the evolution of preclinical and clinical strategies that have improved gene therapy in the eye, showing that treatment of vision loss has a bright future.
authors.affiliation: 1 Department of Ophthalmology and Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
authors.forename: Lolita
authors.initials: L
authors.lastname: Petit
authors.affiliation: 1 Department of Ophthalmology and Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.|2 Department of Neurobiology, University of Massachusetts Medical School , Worcester, Massachusetts.
authors.forename: Hemant
authors.initials: H
authors.lastname: Khanna
authors.affiliation: 1 Department of Ophthalmology and Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.|2 Department of Neurobiology, University of Massachusetts Medical School , Worcester, Massachusetts.
authors.forename: Claudio
authors.initials: C
authors.lastname: Punzo
mesh_terms: D000818:Animals; D005128:Eye Diseases / Q000235:genetics / Q000628:therapy*; D015316:Genetic Therapy; D006801:Humans