Gene therapy for blindness

Researchers in the Children's hospital of Philadelphia from the University of Pennsylvania are developing a new and exciting treatment for blindness. They are mainly researching inheritable retinal degenerations such as Leber congenital amaurosis (LCA). LCA is a disease that begins damaging light receptors in the retina in childhood and well into late adolescence until the patient is blind. The researchers used a vector of a genetically engineered adeno-associated virus to deliver the normal version of the gene RPE65 that is mutated in one form of LCA. So far only 3 patients have received the treatment through a surgical procedure in which the gene is injected into the eye. The patients have reported better sensitivity to light and have been able to navigate an obstacle course better since before the treatment. This is exciting because this is the first time gene therapy has been used to treat a nonlethal pediatric condition. We are still a long way from the movie Gattica but i think that this new breakthrough in gene therapy could pave the way for scientist and researchers to better understand our genetic makeup and the effects they can have in treating it.

http://www.sciencedaily.com/releases/2008/04/080427194726.htm