At the moment LCA is untreatable but a number of novel interventions are currently under investigation including gene therapy.
The aim of gene therapy for the eye is to restore aspects of vision by using a harmless virus to introduce a normal copy of a defective gene into the cells where it is needed to make it available for use, essentially ‘replacing and correcting’ the defect.
Researchers at Moorfields Eye Hospital and UCL Institute of Ophthalmology were some of the first people in the world to conduct gene therapy in the eye for LCA caused by defects in the gene RPE65. This research helped prove that gene therapy might help some people with this particular form of LCA in the future.
More research will need to be undertaken to determine to what extent gene therapy might help people with LCA and to determine what other forms of LCA might benefit from other forms of gene therapy.
Hello, my name is Jake Ternent.
I have Leber congenital amaurosis (LCA). In 2020 and 2021, I received Luxturna gene therapy for this inherited rare eye condition.
I feel very lucky and privileged to be one of the first people in the United Kingdom to receive this ground-breaking treatment, which is hoped to keep my vision around for the rest of my life.
This has improved my life so much by simply just increasing my night vision and increasing my peripheral vision, increasing other facets of my vision. I'm so thankful for Moorfields for this amazing cutting edge treatment, to be available on a magnificent NHS. I feel so privileged and so honoured to receive such an amazing treatment, in the time where we are struggling with what's going on in the world. I feel very lucky and very happy with the outcome, and look forward to seeing more things in the future.
You can self-fund or use private medical insurance to fund your treatment.