Experimental Gene Therapy Restores Vision in Patients with Inherited Blindness

An experimental gene therapy has shown promising results in restoring vision for patients with inherited blindness. College student Olivia Cook, who had limited central vision due to an inherited retinal disorder known as Leber congenital amaurosis, underwent a CRISPR-based gene-editing treatment that significantly improved her vision.

Dr. Jason Comander, director of the Inherited Retinal Disorders Service at Mass Eye and Ear, led the clinical trial that involved 14 volunteers, including Cook. The trial results, published in the New England Journal of Medicine, revealed that the gene-editing tool was associated with a ‘meaningful improvement’ in vision for most patients without serious side effects.

Cook, now 22 and studying marketing at Missouri State University, shared her experience post-surgery, expressing newfound hope for advancements in science and vision restoration. Following the treatment, she noticed significant improvements in her ability to see in dim lighting conditions, a challenge she faced prior to the gene therapy.

Reflecting on a moment with friends on a balcony decorated with Christmas lights, Cook described the profound impact of the treatment. While her right eye could only perceive silhouettes, her left eye, treated with the CRISPR therapy, allowed her to see her friends’ facial features clearly under the dimly lit setting.

Despite the positive outcomes observed in the trial, the therapy is still considered experimental, and further research involving a larger group of individuals is necessary to validate the results. However, the successful restoration of vision in patients like Cook offers hope for the future of treating inherited retinal disorders and combating blindness worldwide.


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