11-year-old becomes first UK patient to receive gene therapy for rare condition

Jump to contentThank you for registeringPlease refresh the page or navigate to another page on the site to be automatically logged inPlease refresh your browser to be logged inAllNewsSportCultureLifestyleCatherine L'Estrange became only the second person in the world to have the treatment (Family handout/PA)Catherine L’Estrange, an 11-year-old from London, has become the first patient in the UK to receive groundbreaking gene therapy for Bardet-Biedl syndrome (BBS), a rare condition causing progressive sight loss. The innovative treatment, performed at St Helier Hospital in March, involved injecting healthy copies of a specific gene directly into her eye's retina to stabilise or potentially improve her vision. Catherine, who was diagnosed with BBS as a baby, hopes the procedure will enable her to continue her favourite hobby of reading books, as patients with BBS typically experience blindness by their early twenties. This pioneering procedure had only been performed on one other individual globally before Catherine, a 17-year-old Canadian girl, and has since been carried out on one other younger child. While early feedback from patients and families has been positive, suggesting improved vision in dim light, surgeons caution that it will take several years to determine the full long-term results of the therapy. In fullGirl, 11, becomes first UK patient to have gene therapy to stop her going blindMore bulletinsThank you for registeringPlease refresh the page or navigate to another page on the site to be automatically logged inPlease refresh your browser to be logged in

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