NEW YORK - "I process the world with my ears and see with my heart and observe it with my soul what most people can see with their eyes," Michael Kalberer tells FOX 5 News.
Kalberer was born with Cerebral Palsy and a rare vision loss disorder called Leber's Congenital Amaurosis. At 12 years old, he was diagnosed as legally blind. Now, at 43, there is a possible light at the end of this dark tunnel.
"I just realized how truly special the opportunity is. I wanted to be a leader for people with inherited retina disease," said Kalberer.
And a leader he is. Michael, who met us near his home in Mineola, was one of the first two participants in a groundbreaking medical trial designed to try and restore vision to patients like him. The study involves the revolutionary gene-editing technique called CRISPR, which allows scientists to make precise changes in DNA. Dr. Eric Pierce, who’s a principal investigator with this trial, calls Michael a pioneer.
"It has the potential to bring genetic therapy to many currently untreatable diseases and we would never be able to get there unless these patients are willing to participate as subjects in these kinds of clinical trials," said Dr. Pierce.
In studies done to date with CRISPR, doctors take cells out of the body, edit them in a lab and then infuse those edited cells back into patients. This is the first time scientists are using CRISPR to edit DNA when it's still inside patients' bodies. The retina, for instance, can’t be removed because it’s attached to the brain. Kalberer got the surgery about nine months ago and is currently being observed.
"I dream about seeing smiles, I dream about seeing my nieces at their wedding," said Kalberer.
Kalberer and his doctors aren’t able to reveal if he has regained any vision yet because they need to wait for the full trial to finish. Researchers expect to share that data by the end of the year.