Gene Therapy Restores Vision

Scientists at the University of Florida Restore Some Sight to Three Adult Patients with Congenital Vision Loss

One year ago the retina of two men and one woman in their 20s were injected with a harmless virus that contained vision correcting genes.  All three patients lived with severe vision loss from a congenital problem call Leber’s Congenital Amaurosis Type 2.  A gene responsible for a necessary protein used in the visual process is defective, gene therapy fixes the failed gene.  One year after the gene therapy treatment these patients reporting being able to see light which was a significant improvement in their vision.  One of the patients reported that she was able to see her parents clock, she had never been able to do this before.  The study has two more years to run and it is likely that more patients will be added.

This is a very exciting medical development and shows that gene therapy holds great promise.  There are so many genetic conditions, not only in the eye but the entire body, that can benefit from this type of treatment.  Much research still needs to be done, however this lays the groundwork for promising future development. The exciting thing about this discovery is that it shows that it is possible to fix bad genes and improve people’s lives.