UMMS Affiliation

Department of Ophthalmology; Gene Therapy Center; UMass Metabolic Network

Publication Date


Document Type



Cellular and Molecular Physiology | Eye Diseases | Molecular Biology | Therapeutics


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.

Rights and Permissions

Copyright Lolita Petit et al., 2016; Published by Mary Ann Liebert, Inc.

DOI of Published Version



Hum Gene Ther. 2016 Aug;27(8):563-79. doi: 10.1089/hum.2016.040. Epub 2016 Jun 13. Link to article on publisher's site

Journal/Book/Conference Title

Human gene therapy

Related Resources

Link to Article in PubMed

PubMed ID


Creative Commons License

Creative Commons Attribution-Noncommercial 4.0 License
This work is licensed under a Creative Commons Attribution-Noncommercial 4.0 License