11-Year-Old Becomes First UK Patient to Receive Gene Therapy for Sight Loss

Catherine L’Estrange, an 11-year-old from North Acton, London, has become the first UK patient to undergo innovative gene therapy aimed at halting sight loss caused by Bardet-Biedl syndrome (BBS). The treatment involves injecting healthy copies of a gene directly into the retina to preserve or potentially improve vision. BBS, affecting approximately one in 100,000 births in the UK, often leads to blindness by the late teens or early 20s. Catherine's procedure was performed at St Helier Hospital, and early feedback from families has been promising, though long-term results will take years to assess.