Gene Therapy Breakthrough

by Dan Roberts April 28, 2008 According to two studies published in the New England Journal of Medicine*, doctors have, for the first time, used gene replacement therapy to restore vision in patients with Leber’s congenital amaurosis. Leber’s syndrome is a form of retinitis pigmentosa (RP) that affects children and often leads to blindness by [Read More]

Gene Therapy Studies Successful in Slowing Neovascularization

by Dan Roberts January 2002 (Updated February 2006) Researchers at Johns Hopkins’ Wilmer Eye Institute have been able to successfully slow blood vessel growth in laboratory mice in two different experiments involving injection of genes, and human trials have begun. In one study (Mori, K., et al., “Inhibition of Choroidal Neovascularization by Intravenous Injection of [Read More]

Gene Therapy Successful in Halting CNV

by Dan Roberts Researchers have been able to halt (at least temporarily) choroidal neovascularization (CNV) with gene replacement therapy. This was done by single injections into the eyeballs of patients during a phase I clinical trial. Here is more detail, as reported on January 17, 2006 by Genetic Engineering News (www.genengnews.com): Clinical Trial on Gene [Read More]

Researchers Restore Vision In an Animal Model of Childhood Blindness

By Tom Hoglund Information Officer, Foundation Fighting Blindness July 2000 In a ground breaking study published in the July issue of The Proceedings of the National Academy of Sciences, researchers rapidly restored lost vision in a mouse model of Leber congenital amaurosis (LCA) using oral doses of a chemical compound derived from vitamin A. LCA [Read More]

Foundation Researchers Restore Vision in Canine Model of Childhood Blindness

by Tom Hoglund In one of the single most important advances in the history of retinal degeneration research, a group of Foundation Fighting Blindness-supported scientists used gene therapy to restore vision in a canine model of severe childhood blindness, known clinically as Leber congenital amaurosis (LCA). This finding, published in the May issue of Nature [Read More]