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Research Field Cell & gene therapy, Neurological

A Fighting Chance

At a Glance

  • New research, the early stage of which was funded by Fight for Sight, shows positive results in protecting sight for patients with a type of LCA
  • The new therapy uses antisense oligonucleotides to repair genetic faults of LCA type 10, targeting a defect in the CEP290 gene
  • Mike Cheetham from the UCL Institute of Ophthalmology led initial research published in 2016, developing retinal organoids using cells derived from LCA10 patients
  • A clinical trial, involving five research organizations around the world, showed improvement in patients’ visual acuity.

At the beginning of this year, I was delighted to see research from a clinical trial published in Nature Medicine (1) that showed promising results in preventing sight loss – or even restoring sight – for patients with a type of Leber congenital amaurosis (LCA). I’m proud that Fight for Sight funded the early stage research that helped enable this finding, which is what our charity is all about.

LCA is a disease of childhood and represents a group of rare inherited disorders that progressively affect the photoreceptors of the retina. People with LCA have severe sight loss at an early age, and it affects around two or three people in every 100,000.

Several different types of LCA have been identified to date – driven by mutations in genes that have been shown to be crucial for normal visual function, including RPE65 (LCA type 2) and CEP290 (LCA type10). Mutations in these genes can result in loss of an essential protein leading to visual impairment. Recently, the first viral gene therapy was approved for LCA type 2; it can improve patient vision in dim light, but there are no therapies for the other more common forms of LCA. And that’s why it is so important that we fund research in this area.

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About the Author

Neil Ebenezer

Director of Research, Policy and Innovation at Fight for Sight, UK

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