Gene Therapy Ideal for Neurodegenerative Eye Disorders

"Diseases of the eye that cause vision loss and blindness, especially neurodegenerative disorders affecting the retina, are ideal targets for gene therapy, including gene replacement and promising corrective gene editing strategies," reports Medical News Today. "A comprehensive Review article providing an overview of emerging therapeutic approaches and innovative gene delivery and gene editing tools to treat ocular diseases is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free for download on the Human Gene Therapy website." Read more.

Gene Therapy Treats Inherited Eye Disease

"What could become the first gene therapy to win approval in the United States moved closer to market on Monday, when its developer announced that the medicine had succeeded in a late-stage clinical trial in treating an inherited eye disease that can cause blindness," according to the New York Times. "The developer, Spark Therapeutics, said the treatment had allowed people with certain so-called inherited retinal dystrophies to more easily maneuver in dimmer light than they could before. The company said it planned to apply to the Food and Drug Administration next year for approval to sell the product. 'We saw substantial restoration of vision in patients who were progressing toward complete blindness,' Dr. Albert M. Maguire, a professor of ophthalmology at the University of Pennsylvania and a lead researcher in the study, said in a news release being issued by Spark." Read more.

A "Fix" for Colorblindness Near at Hand?

Simulation of extreme colorblindness

"More than 10 million Americans have trouble distinguishing red from green or blue from yellow, and there's no treatment for colorblindness." reports National Public Radio (NPR). "A biotech company and two scientists hope to change that. On Wednesday, Avalanche Biotechnologies in Menlo Park and the University of Washington in Seattle announced a licensing agreement to develop the first treatment for colorblindness. The deal brings together a gene therapy technique developed by Avalanche with the expertise of vision researchers at the University of Washington. 'Our goal is to be treating colorblindness in clinical trials in patients in the next one to two years,' says Thomas Chalberg, the founder and CEO of Avalanche." Read more or listen to the story.

Gene Therapy Leading to Treatments for Eye Disease

"Gene therapy strategies to prevent and treat inherited diseases of the retina that can cause blindness have progressed rapidly. Positive results in animal models of human retinal disease continue to emerge, as reported in several articles published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The articles are available free on the Human Gene Therapy website." That's the start of a post on Medical News Today. Read more.

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.)

 Read more.