Gene Therapies Might Treat Inherited Causes of Blindness

"The hope is that similar therapies targeting other mutations can be used to treat a large number of inherited diseases that cause blindness," according to a WebMD post. "Katherine A. High, MD, of Children’s Hospital of Philadelphia, who also worked on the study, says there are now 200 known genetic mutations that cause vision loss. Because LCA is a degenerative disease, there is also hope that the treatment may one day be used in very young children, or even babies, before vision loss has occurred." Read more.

This is how Medical News Today wrote about the event: "Researchers from the Perelman School of Medicine at the University of Pennsylvania and The Children's Hospital of Philadelphia have conducted a recent study, published in Science Translational Medicine which focuses on gene therapy for congenital blindness. The scientists were able to improve sight in 3 adult patients who had previously been treated in one eye. The researchers used the same treatment on the second eye of the patients, and they were able to see in low-light situations and also find their way around. There were no conflicting effects reported." Read more.


Three years ago Scientific American reported: 

Gene therapy has been rhapsodized and vilified in its nearly two decades of human testing, helping some and making others sicker. But a new 12-month clinical trial has shown that, at least in one ocular disease, it appears safe and—perhaps even more impressive—effective.

The research, part of a phase I clinical trial to test the safety of the treatment, was published as a letter to the editor in The New England Journal of Medicine earlier this week and will be in the September issue of Human Gene Therapy. (The paper was co-authored by about a dozen researchers, two of whom own equity in a company that could profit from a commercialized version of this procedure.)

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