Developing gene therapies for corneal endothelial disorders

Abstract

AbstractTechniques of gene transfer to corneal cells have been in development for at least 15 years. Studies on feasibility of gene transfer and kinetics of transgene expression have been undertaken ex vivo and in vivo using physical, viral vector and non‐viral vector techniques of gene transfer. Functional gene transfer research has been developed for applications such as anti‐angiogenesis, modulation of stromal wound healing or endothelial cell cycle control and prevention of allotransplant rejection. The absence of such application in inherited disorders of corneal endothelium points to absence of in vivo models for study and the availability of surgical treatment which, in many patients, is effective. Opportunities in the field of gene‐based approaches to disorders of corneal endothelium lie in newer designs of low‐immunogenicity vectors, longer duration of transgene expression and application in eye banking. Experimental approaches which in essence use cDNA as an alternative to protein or drug treatment will be discussed. Supported by NIHR Biomedical Research Centre in Ophthalmology, Moorfields Eye Hospital & UCL Institute of Ophthalmology, London, United Kingdom