Gene therapy clinical trial. A researcher selects a vial containing gene therapy treatment for Leber's congenital amaurosis (LCA), a rare inherited ey


Gene therapy clinical trial. A researcher selects a vial containing gene therapy treatment for Leber's congenital amaurosis (LCA), a rare inherited eye disease. The condition is caused by a defective gene thought to result in abnormal development of photoreceptor cells in the retina. In clinical trials, patients with a mutation in the RPE65 gene were successfully treated using adeno-associated virus as a vector to deliver healthy genes to retinal cells, restoring vision. Photographed at Nantes University Hospital, France.


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