Gene therapy for inherited blindness shows promise in first clinical trialBMJ 2014; 348 doi: https://doi.org/10.1136/bmj.g269 (Published 16 January 2014) Cite this as: BMJ 2014;348:g269
- Ingrid Torjesen
The first phase I clinical trial of gene therapy for an inherited cause of blindness has shown sustained improvements in visual acuity.
In the trial, funded by the Department of Health for England and the Wellcome Trust, six patients had genes injected into the retinal cells of one eye, with no adverse effects. Six months later all the patients reported improvements in their vision in dim light, and two of the six, who have now been followed up for up to two years, were able to read more lines on an eye chart.
The six patients who received the gene therapy had choroideraemia, a rare inherited blindness that affects one in 50 000 people and for which there is no treatment.
A third of eye disorders have a …