New study on promise of gene therapy for Leber congenital amaurosis

Leber congenital amaurosis (LCA) is a congenital retinal dystrophy present in approximately 1 of 80,000 births. It is estimated that about 3,000 people in the United States are living with LCA and will likely become blind in their lifetimes. Recently, there has been progress in gene therapy for this condition. In a recent study published in the current issue of the Journal of AAPOS, the Official Publication of the American Association of Pediatric Ophthalmology and Strabismus, researchers from the University of Iowa Hospitals and Clinics, Iowa City, and The Hospital for Sick Children, Toronto, found that only a limited number of patients possess the exact genetic mutation necessary for a positive outcome from the therapy.

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