Dr. Thomas Mendel from Ohio State University Awarded $69,266 Knights Templar Eye Foundation Grant for Batten Disease Research

Dr. Thomas Mendel from the Ohio State University Wexner Medical Center was awarded a grant for $69,266 for his research entitled: Gene Therapy for Pediatric Retinopathy from Batten Disease

Batten disease is one of the most common and devastating diseases of the brain and nervous system in children. This family of diseases is caused by mutations in 1 of 13 genes that normally allow nerve cells to break down waste products. Without the ability to breakdown cellular waste, those nerve cells become diseased and lead to rapid blindness, coordination and strength loss, decline in intelligence, and eventually seizures and premature death, usually before a child is 10 years old.

Thankfully, there has been some promising success by treating some patients with gene therapy in their spinal columns, helping to insert a working copy of the gene into the neural cells in the brain that are naturally bathed in spinal fluid. However this gene therapy isn’t yet reaching the eye following injection in the spinal column, so the patients are still going blind.

This project aims to determine how best to deliver the gene therapy to the eye and the tissue in the back of the eye that turns light into a nerve signal, the retina. First Dr. Mendel will look at if he should inject the gene in front of or behind the retina with different surgical techniques. Second and very importantly, he will monitor closely for not only gene function, but also any signs of inflammation.