Document Type
Article
Publication Date
2013
Abstract
Corneal blindness is the third leading cause of blindness worldwide. Gene therapy is an emerging technology for corneal blindness due to the accessibility and immune-privileged nature of the cornea, ease of vector administration and visual monitoring, and ability to perform frequent noninvasive corneal assessment. Vision restoration by gene therapy is contingent upon vector and mode of therapeutic gene introduction into targeted cells/tissues. Numerous efficacious vectors, delivery techniques, and approaches have evolved in last decade for developing gene-based interventions for corneal diseases. Maximizing the potential benefits of gene therapy requires efficient and sustained therapeutic gene expression in target cells, low toxicity, and a high safety profile. This review describes the basic science associated with many gene therapy vectors and the present progress of gene therapy carried out for various ocular surface disorders and diseases.
Recommended Citation
Mohan RR, Rodier JT, Sharma A. Corneal Gene Therapy: Basic Science and Translational Perspective. Ocul Surf. 2013;11(3):150-164. doi:10.1016/j.jtos.2012.10.004.
Copyright
Elsevier
Creative Commons License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.
Included in
Musculoskeletal, Neural, and Ocular Physiology Commons, Ophthalmology Commons, Other Pharmacy and Pharmaceutical Sciences Commons
Comments
NOTICE: this is the author’s version of a work that was accepted for publication in Ocular Surface. Changes resulting from the publishing process, such as peer review, editing, corrections, structural formatting, and other quality control mechanisms may not be reflected in this document. Changes may have been made to this work since it was submitted for publication. A definitive version was subsequently published in Ocular Surface, volume 11, issue 3, in 2013. DOI: 10.1016/j.jtos.2012.10.004
The Creative Commons license below applies only to this version of the article.