Treating vision with gene therapy

SGGP 07/27/2023 08:56

Teenager Antonio has regained his sight after years of living in the dark thanks to gene therapy eye drops in the US.

Gene Therapy Vision Treatment at Bascom Palmer Eye Institute

This is promising news for the millions of people who suffer from eye diseases. Antonio was born with a rare genetic condition that causes blisters all over his body and eyes. The disease affects about 3,000 people worldwide . Antonio's condition stems from a mutation in a gene that produces the protein collagen 7.

In August 2022, Antonio had surgery on his right eye, and then Dr. Alfonso Sabater at the Bascom Palmer Eye Institute in the US conducted an experimental treatment with gene therapy eye drops based on a treatment called Vyjuvek, which uses an inactivated herpes simplex virus to create active copies of that gene. This line of eye drops was approved by the US Food and Drug Administration (FDA) for treatment after 2 years of research and testing.

Before treatment, Antonio’s eyes were covered with cloudy scars. After treatment with gene therapy eye drops, the scars disappeared. Antonio’s vision in his right eye is 20/25. His left eye has vision of nearly 20/50. Antonio’s skin has also improved after participating in the clinical trial.