Scientists say gene therapy might bring back eyesight to kids and grown-ups who lost it due to a rare inherited problem called Leber congenital amaurosis. This disease happens because of changes in the GUCY2D gene, which plays a key role in making proteins that allow us to see. People with this type of Leber congenital amaurosis (LCA1) go blind when they’re young.
However, scientists at the University of Pennsylvania claim that a new gene therapy shot under the retinas of kids and adults with LCA1 made their vision 100 to 10,000 times better. The study results came out Sept. 5 in The Lancet journal.
The new trial involved 15 LCA1 patients in total — 12 adults and three kids. All of these participants had eyesight equal to or below 20/80 which indicates these participants were able to see at 20 feet distance, similar to normal vision a person can see at 80 feet distance.Â
Glasses didn’t help much, since they fix the eye’s focus, not the root medical cause behind the vision loss. Atsena Therapeutics created and funded a new gene therapy called ATSN-101. This small phase 2 trial aimed to check the treatment’s safety and look at how different doses work. Doctors injected the therapy at three different levels. Three grown-ups got low, medium, and high doses of ATSN-101. After making sure it was safe, six people (including three kids) got high-dose ATSN-101.
The research group says all patients’ sight got better fast within a month after treatment. These improvements will stick around for at least a year. Two out of nine patients who received the high-dose treatment showed a 10,000-fold improvement in their vision, according to Cideciyan’s team.
Side effects were possible, and stemmed from the surgery used to implant the gene therapy. These included small bleeds within the eye that healed later, or short-term eye inflammation, which doctors treated with steroids.
“Our latest clinical trials and past experience have an influence on treatment success giving us hope to treat 20 percent of infantile blindness caused by inherited retinal problems,” said Dr. Tomas Aleman in a UPenn news release. He’s a professor in ophthalmology. “We now aim to improve treatments and address early signs of these conditions once we confirm safety,” he added. “We expect similar methods will result in good outcomes for other types of congenital retinal blindness.”
Reference:Â
Gene therapy reverses a rare cause of vision loss – drugs.com MedNews
