Researchers at UCL Institute of Ophthalmology, Moorfields Eye Hospital and the NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology have recently completed a first-in-human trial (ClinicalTrials.gov NCT00643747) of gene therapy for an inherited form of blindness. Children with this condition lack night vision and lose daylight vision.
The results of the trial are published in NEJM. Professor James Bainbridge, lead clinician said, "this study confirms our preliminary findings (published in NEJM, 2008) that gene therapy can improve night vision, providing further evidence of benefit in inherited blindness".
Professor Robin Ali who lead the research group commented, "our latest results provide confirmation of efficacy but the data, together with results of a parallel study in dogs, indicate that the demand for RPE65 is not fully met with the current generation of vectors. We have concluded that early intervention using a more potent vector, expressing higher levels of RPE65 is likely to provide greater benefit and protection against progressive degeneration".
The group has now developed a new, more powerful gene therapy vector and is aiming to test this in a second clinical trial funded by The UK Medical Research Council.
Study: Long-Term Effect of Gene Therapy on Leber's Congenital Amaurosis, James W.B. Bainbridge, Ph.D., et al., NEJM, DOI: 10.1056/NEJMoa1414221, published 4 May 2015.