Gene therapy could treat blindness

A Phase I/II clinical trial has been initiated to evaluate an innovative gene therapy in patients with progressive deterioration in vision caused by an abnormality in the RPE65 gene.

Researchers from University College London's (UCL) Institute of Ophthalmology, Moorfields Eye Hospital, London, UK and Targeted Genetics Corporation will evaluate the effectiveness of an adeno-associated virus (AAV) vector to deliver a normal copy of the RPE65 gene into the cells of the retina. Research in animal models has demonstrated that the AAV-mediated delivery can improve and preserve vision.

The trial, which is the first of its kind and is being funded by the UK Department of Health, involves adults and children who have progressive vision deterioration caused by the genetic defect.

The purpose of this study is to evaluate the safety and efficacy in human patients.