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About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a paper published in the New England Journal of Medicine.
Gene therapy has shown promise in improving eyesight in congenitally blind children with Leber Congenital Amaurosis (LCA), a rare disease.
For her entire life, college student Olivia Cook had only a small degree of central vision. It was as if she was watching the world through a straw hole, and in dimly lit places, she could not make out people’s faces, only their silhouettes.
Key Takeaways
An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says.