Baby Is Healed With World’s First Personalized Gene-Editing Treatment
The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases.
Something was very wrong with Kyle and Nicole Muldoon’s baby.
The doctors speculated. Maybe it was meningitis? Maybe sepsis?
They got an answer when KJ was only a week old. He had a rare genetic disorder, CPS1 deficiency, that affects just one in 1.3 million babies. If he survived, he would have severe mental and developmental delays and would eventually need a liver transplant. But half of all babies with the disorder die in the first week of life.
Doctors at Children’s Hospital of Philadelphia offered the Muldoons comfort care for their baby, a chance to forgo aggressive treatments in the face of a grim prognosis…
