Gene therapy helps the blind to see, play softball

People with Leber’s congenital amaurosis (LCA), a rare eye disease,  are blind from birth. A new gene therapy trial conducted by the University of Pennsylvania and The Children’s Hospital of Philadelphia, however, improved vision in subjects with a single injection. The results were most effective in children, who had the benefit of less accumulated retinal damage when compared to the adults.

Starting two weeks after the injections, all 12 subjects reported improved vision in dimly lit environments in the injected eye. An objective measurement, which measures how the eye’s pupil constricts, showed that all the subjects were able to detect significantly more light after treatment and also showed greater light sensitivity in each patient’s treated eye compared to the untreated eye. In addition, before treatment, nine patients had nystagmus, an involuntary movement of the eyes that is common in LCA. After treatment, seven of them had significant improvements in nystagmus.

Among the success stories was a kid who went from needing assistance to see the front of his classroom to being able to play softball for the first time, which is pretty incredible and pretty cool.

EDIT: Cleaned up some language in the first paragraph.