Baby is cured of the first treatment of personalized genes in the world

Something was wrong with Kyle’s baby and Nicole Muldoon.

Doctors have speculated. It may be meningitis? Maybe seticemia?

They obtained an answer when KJ had only one week. It had a rare genetic disorder, a deficiency in CPS1, which affects only one in 1.3 million babies. If he survived, he would have serious mental delays and development and would finally need a liver transplant. But half of all babies with trouble died in the first week of life.

The doctors of the Philadelphia Children’s Hospital offered Comfort Care Muldoons for their baby, a chance to give up aggressive treatments in the face of a dark prognosis.

“We liked him and we didn’t want him to suffer,” said Ms. Muldoon. But she and her husband decided to give KJ a chance.

Instead, KJ has made medical history. The baby, now 9 and a half months old, has become the first patient of all ages to undergo personalized gene editing treatment, according to his doctors. He received a infusion made for him and designed to repair his precise mutation.

Investigators who led the effort to save KJ present their work Thursday at the annual meeting of the American Society of Cell & Gene Therapy and also publish it in the New England Journal of Medicine.