Gene Therapy Shows Promise for Blindness

Scientists for the first time have used gene therapy to dramatically improve sight in people with a rare form of blindness.


According to Blindness.Com website, the new advance in gene therapy is providing hope for people with a debilitating retinal disease known as achromatopsia or day blindness. Researchers from the University of Pennsylvania have used gene therapy to successfully treat dogs with the same condition.


The study’s lead investigators, Andras Komaromy, D.V.M., Ph.D., and Gustavo Aguirre, V.M.D., Ph.D., presented the results of their FFB-funded achromatopsia study in April 2008 at the annual conference of the Association for Research in Vision and Ophthalmology.


The investigators’ ultimate goal is to move the gene therapy into human studies, though the treatment’s safety and long-term effectiveness need to be ensured through more preclinical work before the treatment can be moved into the clinic.