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Gene Therapy Provides Vision to People who Were Nearly Blind
Date:4/27/2008

he success in the first three patients, however, will position the researchers well to plan Phase II clinical studies to evaluate the treatment's potential effectiveness in younger children who were born blind from LCA. The investigators believe the treatment has the potential to give near-normal vision to these children.

The first step toward the development of this treatment began with the discovery of the RPE65 gene in 1993. In 2000, the first dog born blind from LCA, a Briard named Lancelot, was successfully treated with gene therapy, and has been seeing well since then with just a single treatment. More than 50 dogs have now been treated successfully and are all seeing well. Clinical trials of the procedure began in October 2007 at the Foundation-funded Children's Hospital of Philadelphia (CHOP)-Penn Pediatric Center for Retinal Degenerations in Philadelphia.

More than 10 million people across the United States are affected by retinal degenerative diseases which include: macular degeneration, retinitis pigmentosa, and Usher syndrome.

About the Foundation Fighting Blindness

The Foundation Fighting Blindness (http://www.FightBlindness.org) is the largest source of non-governmental funding for retinal degenerative disease research in the world. The urgent mission of the Foundation Fighting Blindness is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome, and the entire spectrum of retinal degenerative diseases.


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SOURCE Foundation Fighting Blindness
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