Sight-saving gene therapy treatment could change lives

A gene therapy treatment has enabled sight improvements for blind infants, through pioneering work by University College London’s (UCL) Institute of Ophthalmology and Moorfields Eye Hospital.  

Children born with a rare genetic deficiency, affecting the AIPL1 gene, who were blind from birth, have successfully undergone treatment at Great Ormond Street Hospital to revive dying retinal cells.  

The procedure involved injecting healthy copies of the gene encased in a harmless virus into the retina through keyhole surgery, replacing the defective gene.  

All four children participating in the trials experienced sight improvements in the eye treated in the following three to four years, compared to their untreated eye that continued to deteriorate. Due to health and safety risks, only one eye was given the experimental medicine.