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An American living with Leber congenital amaurosis 10 (LCA10) has become the first clinical trial participant in the world to receive a CRISPR gene editing treatment in vivo (inside the body).
Progress towards treatments for inherited retinal conditions continues to gather pace and there’s been lots going on in the last few months, with more and more approaches being explored. This round-up gives a flavour of the variety of developments, including plenty that are not specific to a particular genetic fault.
We provide funding for innovative, high quality research projects investigating the causes and potential treatments for all forms of inherited retinal disease.
A discovery made as part of research funded by Retina UK has led to a prestigious award of $2.5 million, enabling scientists to undertake more in-depth investigations and work towards a treatment for a particular type of retinitis pigmentosa.
Stem cells are a special type of cell, which under the right conditions can be encouraged to grow into any other type of cell in the body, including retinal cells (rods, cones and retinal pigment epithelial cells).
When we talk about gene therapy we are usually referring to use of a harmless virus, called a vector, to deliver a normal copy of a defective gene into the cells of the eye.
Our Keynote speaker will be Mr Kanmin Xue. Mr Xue is a Consultant Vitreo-retinal Surgeon at the John Radcliffe Hospital and Wellcome Trust clinician scientist fellow at the University of Oxford where he leads the Retinal Disease and Repair Group.
“All the promising research makes you feel like there is light at the end of that very dark tunnel after all.”
Growth factors are substances that promote the health and function of cells and tissues in the body.
ProQR has published the top-line results from its Phase 2/3 “Illuminate” trial of the RNA therapy sepofarsen for Leber congenital amaurosis type 10 caused by a specific mutation in the CEP290 gene.
