Nearby Chester county-based anesthesiologist Nicole Henwood has devoted her life to funding gene therapy research with the hope of finding a cure for her nine-year-old son, A.J., writes Sandy Bauers for The Philadelphia Inquirer.
A.J. has a rare disease known as NF2, or neurofibromatosis Type 2, for which there is no cure.
To help change that, his mother, who works at Premier Orthopedics, has founded the nonprofit, NF2 BioSolutions. Its goal is to accelerate gene therapy research for NF2.
The disease causes tumors to grow in the patient’s body, mostly in the nervous system. This makes affected people lose their ability to hear, walk, talk, and see, among other things.
In addition to there being no cure, surgery is also not an option as the tumors grow into the nerves. Now, though, Henwood hopes that gene therapy could offer a brighter future for her son.
“In some diseases, like diabetes or hypertension, many genes are involved,” said Henwood. “But with this, it’s just one gene that is mutated. If we could fix that one, we could essentially stop the disease.”
Read more about NF2 BioSolutions in The Philadelphia Inquirer here.