A groundbreaking gene therapy trial at Moorfields Eye Hospital in London has resulted in “life-changing improvements” for a young Connecticut boy and four other children suffering from a severe form of childhood blindness. Jace, 4, who was legally blind from birth and could barely distinguish between light and dark, now has much better vision.
The toddler’s rapid vision loss was caused by a rare genetic condition. When Jace was only a few months old, his parents, DJ and Brendan, noticed something was amiss among his peers. “Around 8 weeks old, when babies should start looking at you and smiling, Jace wasn’t doing that yet,” DJ told the BBC.
The most recent advances come from inserting healthy copies of a faulty gene directly into the back of an eyeball, addressing a severe form of the disease. Following an eye consultation, the family decided to participate in the pioneering trial across the pond.
“You never think it’s going to happen to you, of course, but there was a lot of comfort and relief to finally find out… because it gave us a way to move forward,” Brendan told me. However, DJ stated that Jace’s operation was quick and “pretty easy.” The youngster had a few nicks in his eye, when they injected the healthy gene into the retina using a minimally invasive procedure.
In just one month, the youngster’s vision improved significantly. “After the operation, Jace was already spinning, dancing, and making the nurses laugh.
He began responding to the TV and phone within a few weeks of surgery and, within six months, could recognize and name his favorite cars from several meters away; however, it took his brain time to understand what he could now see,” DJ explained.
“Prior to surgery, we could have held up an object near his face and he would have been unable to track it at all. Brendan said, “Now he’s picking things off the floor, hauling out toys, and doing things he wouldn’t have done before because of his sight.”
His parents stated that this may not be the final treatment he will require in his life, but the progress he has made thus far is assisting him in better understanding the world.