Gene Therapy Successfully Restores Cone Function in Colorblind Children
The study, which took advantage of the plasticity of the emerging neural networks in the developing teenage brain, provides optimism that the therapy is successfully reactivating brain-retinal communication channels that had remained dormant. Academic research has been conducted to determine whether the medicine is changing the brain circuits specific to the cones in conjunction with a phase 1/2 clinical study in children with achromatopsia. Achromatopsia is caused by one or a few genes having disease-causing mutations. It has an impact on cone cells, one of the two types of photoreceptors in the eyes (the other being rods). Because cones are crucial for color vision, people with achromatopsia are completely colorblind in addition to having very poor overall vision and finding bright light painful (photophobia). Since the dormant cone cells exist but do not send messages to the brain, researchers are working to revive them. "Our study is the first to directly confirm popular belie...