Gene Therapy Clinical Trials For Childhood Blindness

[kiki06]

Clinical trials for the treatment of childhood blindness have begun using gene therapy.

This is the first gene therapy treatment for blindness, targeted specifically in children and has been announced by researchers at UCL (University College London).

The trial will be funded by the Department of Health, and is the first of its kind and could have a significant impact on future treatments for eye disease.

The gene therapy will target children who have a condition known as Leber's congenital amaurosis (LCA). LCA is an inherited retinal degeneration condition which causes progressive deterioration in vision, due to an abnormality in a particular gene called RPE65.

The defect in RPE65 prevents the normal functioning of the retina, which is important in the light-sensitive layer of cells at the back of the eye. The result is severely impaired vision from a very young age and currently no effective treatments available.


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