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Prof Mariya Moosajee at Moorfields Eye Hospital has asked us to share the message below about the clinical trial she is running. This trial is for a treatment that targets a particular section of the USH2A gene.
Choroideremia is a rare genetic condition resulting in progressive sight loss in men.
The team have found the cause of disease in the first ever family tree drawn up at Moorfields Eye Hospital over 35 years ago, which had remained unsolved until now.
Thank you to all those who completed our recent Sight Loss Survey – almost 700 of you took the time to share your experiences.
Our community is informed and knowledgeable about current research projects into the cause(s) of and treatments for these conditions.
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).
Autoimmune diseases are quite distinct from inherited retinal dystrophies.
As a coalition of sight loss charities, we are calling on the Government to make the benefit system and employment support fit for purpose for blind and partially sighted people.
With all the interest in exciting gene and cell therapies for inherited sight loss, it’s easy to forget that more traditional drugs can also hold promise.
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.
