Gene Editing Miracle: Baby Cured of Life-Threatening Disorder

A 9-month-old baby, KJ Muldoon, was born with a rare disorder causing dangerous ammonia levels in his blood. This condition, if left untreated, can lead to brain damage or death. About 50% of babies with this condition pass away in the first week of life. Doctors at the Children's Hospital of Philadelphia took a revolutionary approach, using customized gene editing to correct a single genetic error in KJ's DNA. "It's all been a miracle," said Nicole Muldoon, KJ's mother, in an interview. After six months of development, KJ received his first infusion in February. The next day, his parents reported that he was "up, laughing, looking around, and playing with his toys." While it's still early to determine the full impact, early signs show benefit. This successful treatment opens the door for treating other genetic diseases like cystic fibrosis, sickle cell, and Huntington's.