Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

TLDR

Retinal gene therapy for Leber's congenital amaurosis, an autosomal recessive childhood blindness, has been widely considered safe and efficacious. Here we describe long‑term follow‑up data from three treated patients. Three years after therapy, vision improvement persisted while photoreceptor loss in the treated retina matched that of the untreated retina, and topographic sensitivity maps up to nearly six years post‑therapy show progressive loss of the previously improved visual fields. Funded by the National Eye Institute; ClinicalTrials.gov number, NCT00481546.

Abstract

Retinal gene therapy for Leber's congenital amaurosis, an autosomal recessive childhood blindness, has been widely considered to be safe and efficacious. Three years after therapy, improvement in vision was maintained, but the rate of loss of photoreceptors in the treated retina was the same as that in the untreated retina. Here we describe long-term follow-up data from three treated patients. Topographic maps of visual sensitivity in treated regions, nearly 6 years after therapy for two of the patients and 4.5 years after therapy for the third patient, indicate progressive diminution of the areas of improved vision. (Funded by the National Eye Institute; ClinicalTrials.gov number, NCT00481546.).