Researchers from the University College London (UCL) Institute of Ophthalmology have reportedly been able to investigate the CEP290 gene fault in leber congenital amaurosis (LCA) retinal cells for the first time.
According to UCL, LCA is a group of disorders that make up 5 percent of all inherited retinal dystrophies where photoreceptor cells in the retina degenerate and die, causing vision loss or blindness. About one in four people with LCA have faults in the CEP290 gene, which up until now scientists did not know why.
In addition to investigating the CEP290-LCA gene, UCL researchers have reportedly tested a promising new way to treat CEP290-LCA with an engineered molecule — antisense morpholino — designed to target the LCA genetic fault.
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Source: University College London