Gene Therapy

We are working with the world’s leading researchers studying gene transfer approaches to pediatric orphan diseases. This groundbreaking and innovative approach employs the AAV9 virus, which is not known to cause adverse effects in humans and is able to deliver a replacement CLN6 gene to the brain.  The malfunctioning of the mutated CLN6 gene is what causes Batten disease, and the replacement gene appears to help restore the mutated gene’s functionality, allowing cells to purge built-up wastes and restore balance in the brain. A first of its kind clinical trial has begun at Nationwide Children’s Hospital to evaluate this investigative treatment, providing hope for children with Batten disease all around the world.