A groundbreaking gene therapy has shown promising results in restoring vision in patients with Leber congenital amaurosis type I (LCA1), a rare genetic condition that causes blindness. In a small trial, those receiving the highest dose of the therapy saw up to a 10,000-fold improvement in light sensitivity and significant gains in reading and navigation abilities.
The therapy uses a virus-based system to deliver a functioning gene into the retina’s light-sensitive cells. The results show promise for expanding this treatment, with further trials planned to confirm safety and efficacy.
Patients who received the highest dose of the therapy saw up to a 10,000-fold improvement in their light sensitivity, were able to read more lines on an eye chart, and improved in their ability to navigate a standardized maze. For many patients, it was akin to finally turning on dim lights after trying to navigate their homes in the pitch black for years.
The trial also tested the safety profile of the treatment, with side effects largely limited to minor surgical complications. The gene therapy itself caused mild inflammation that was treated with steroids.
This breakthrough has paved the way for advancing the therapy in a phase 3 clinical trial and eventually commercializing it. Researchers are hopeful that this treatment will provide meaningful improvements to the quality of life for patients with LCA1, who have severely impaired vision that makes it difficult or impossible to drive, read, or navigate the world visually.
The study was conducted by University of Florida researchers, led by Shannon Boye, Ph.D., and published in The Lancet.
Source: https://neurosciencenews.com/gene-therapy-vision-27621/