Home » Gene therapy eye drops restore sight to a blind 13-year-old suffering from a rare disease

Gene therapy eye drops restore sight to a blind 13-year-old suffering from a rare disease

by admin
Gene therapy eye drops restore sight to a blind 13-year-old suffering from a rare disease

“13-Year-Old Restores Sight with Gene Therapy Eye Drops”

A groundbreaking gene therapy in the form of eye drops has restored sight to a 13-year-old boy who was blind due to a rare disease. The boy, who had already undergone several surgeries, suffered from recessive dystrophic epidermolysis bullosa (DEB), a condition characterized by skin that tears easily and forms blisters. In more than a quarter of patients with DEB, the disease also affects the eyes, leading to wounds or scarring that impairs vision.

The gene therapy, known as beremagene geperpavec (B-VEC), uses a vector virus to deliver a healthy version of the affected gene to cells. Initially conceived as a gel formulation, the therapy was designed to be applied to skin wounds. The 13-year-old boy had previously participated in a clinical trial and responded well to the gel gene therapy in treating his skin wounds. Based on his positive response, researchers developed an eye drop formulation of the gene therapy for the boy’s eyes.

After undergoing preclinical testing and obtaining clearance from the FDA, the boy received B-VEC eye drops. Eight months after treatment, the boy’s right eye showed substantial improvement in vision, so much so that he was no longer considered legally blind. The success of the gene therapy gives hope for future applications on other patients affected by the rare disease.

The case represents a significant milestone in gene therapy and its potential to treat various genetic disorders. The remarkable improvement observed in the boy’s vision serves as a beacon of hope for those suffering from similar conditions.

See also  Expert Müller at the SZ Health Forum: “The weight loss products are getting even better” - Health

The article is taken from “The Messenger”.

You may also like

Leave a Comment

This site uses Akismet to reduce spam. Learn how your comment data is processed.

This website uses cookies to improve your experience. We'll assume you're ok with this, but you can opt-out if you wish. Accept Read More

Privacy & Cookies Policy