Eyeblaster

 Eyeblaster  

Gene therapy for congenital blindness has taken another step forward, as scientists further improved vision in three adult patients previously treated in one eye. .

Gene therapy for congenital blindness has taken another step forward, as scientists further improved vision in three adult patients previously treated in one eye. After receiving the same treatment in their other eye, the patients became better able to see in dim light, and two were able to navigate obstacles in low-light situations. No adverse effects occurred.

Neither the first treatment nor the readministered treatment triggered an immune reaction that cancelled the benefits of the inserted genes, as has occurred in human trials of gene therapy for other diseases. The current research targeted Leber congenital amaurosis (LCA), a retinal eyeblaster disease that progresses to total blindness by adulthood.

Researchers from The Children’s Hospital of Philadelphia Research Institute and from the Perelman School of Medicine at the University of Pennsylvania led the study, published today in Science Translational Medicine.