University of Oxford

Treating blindness using gene-therapy


Robert MacLaren is Professor of Ophthalmology at the University of Oxford.

His work has led the way in treating blindness using gene-therapy, as well as developing new techniques for cataract and retinal surgery.

In 2014 he co-founded Nightstar, a University of Oxford spin-out company established to develop gene therapy for choroideremia, a genetic condition that leads to gradual sight loss. His team successfully completed of the world's first gene therapy trial for choroideremia in 2018.

In 2017, he carried out the first in man gene therapy clinical trial for X-linked retinitis pigmentosa in collaboration with Nightstar. The treatment involved creating a correct version of the faulty gene that causes retinitis pigmentosa and inserting it directly into the cells of the patient’s retina using a virus.

Earlier this year he performed the first gene therapy treatment for age-related macular degeneration (AMD), injecting a gene into the back of the patient’s eye to prevent light-sensitive cells from dying. AMD is the most common form of blindness in the Western world.