KJ Muldoon of Clifton Heights, once a critically ill baby, is “growing and thriving” after a custom-made gene editing treatment, according to gene therapy expert Dr. Rebecca Ahrens-Nicklas.
The 9-1/2-month-old is among the first to have a genetic disease treated through personalized gene editing, according to an Associated Press article appearing in The Keystone.
A small genetic code error created a rare disease known as severe CPS1 deficiency.
It deprived KJ of an enzyme needed to remove ammonia from the body, which builds up and becomes toxic.
The usual treatment is a liver transplant.
His parents, Kyle and Nicole Muldoon weighed their options of an invasive liver transplant or “something that’s never been done before” and chose the treatment.
Since the genetic error was treated, KJ has been able to eat more normally and is recovering well from illnesses like colds. He also needs less medication.
Researchers caution, however, that they will need to study KJ for years.
“We’re still very much in the early stages of understanding what this medication may have done for KJ,” Ahrens-Nicklas said. “But every day, he’s showing us signs that he’s growing and thriving.”
Read more about the impact of KJ’s treatment in The Keystone.
