A single jab of genes that produce light-sensitive pigments can make children with congenital blindness see, according to a new study.
Taking the next step towards using gene therapy to cure disease, the study conducted by researchers from the University of Pennsylvania School of Medicine and the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, showed notable improvement in children with congenital blindness.
For the study, the researchers used gene therapy to safely improve vision in five children and seven adults with Leber’s Congenital Amaurosis (LCA). The greatest improvements occurred in the children, all of whom are now able to navigate a low-light obstacle course-a result that the researchers call “spectacular.” “This result is an exciting one for the entire field of gene therapy. This study reports dramatic results in restoring vision to patients who previously had no options for treatment. These findings may expedite development of gene therapy for more common retinal diseases, such as age-related macular degeneration,” The Lancet quoted Dr. Katherine A. High, co-first author of the study as saying.
Although the patients did not attain normal eyesight, half of them (six of 12) improved enough that they might no longer be classified as legally blind. “The clinical benefits have persisted for nearly two years since the first subjects were treated with injections of therapeutic genes into their retinas,” said senior author Dr. Jean Bennett.
For Bennett, the results build on nearly 20 years of gene studies on hereditary blindness, starting with pioneering work in mice and dogs. “These remarkable results have laid a foundation for applying gene therapy not only to other forms of childhood-onset retinal disease, but also to more common retinal degenerations,” she added.
“Children who were treated with gene therapy are now able to walk and play just like any normally sighted child. They can also carry out classroom activities without visual aids,” said co-first author Dr. Albert M. Maguire.
The study was published in The Lancet.