CRISPR Gene Editing Shows Promise: Early Trial Improves Vision in Blind Children [Video]

An early-stage clinical experiment published in the New England Journal of Medicine found that gene therapy can improve hereditary blindness.

Researchers from the Children’s Hospital of Philadelphia and the Mass Eye and Ear Ocular Genomics Institute experimented with those with Leber Congenital Amaurosis (LCA). This rare hereditary disorder causes eyesight loss from birth. According to a report from Star Local Media, eleven of 14 subjects who got a single gene-editing injection improved their eyesight in one eye.

Dr. Tomas Aleman, the pediatric ophthalmologist for the study, described it as remarkable because it was the first time congenitally blind children received gene editing treatment to enhance their daytime vision.

Lead researcher Dr. Eric Pierce stressed the therapy’s tremendous impact, especially for those without previous treatment alternatives. He emphasized participants’ encouraging remarks, including newfound skills like recognizing food on their plates.

Life-Changing Results

LCA, usually occurring at birth or within six months, blinds two …