KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week.
KJ has spent the majority of his life at Children’s Hospital of Philadelphia after being diagnosed with a one-in-a-million, deadly genetic disease shortly after birth.
Wearing a cap and gown to symbolize his “graduation” from the hospital, baby KJ was discharged home to his parents and siblings on Tuesday after spending 307 days at Children’s Hospital of Philadelphia.
KJ “had the most severe variant,” Dr. Ahrens-Nicklas, one of KJ’s doctors at Children’s Hospital of Philadelphia, told ABC News.
CRISPR gene-editing technology was the perfect tool for a rare genetic disease like KJ’s, and potentially future babies born with slightly different genetic errors.
KJ Muldoon, a 10-month-old infant who made national news after being given a novel gene-editing treatment, was discharged from the hospital this week.
Shortly after birth, KJ was diagnosed with a rare and fatal genetic disease, and he has spent most of his life at Children’s Hospital of Philadelphia. His doctors worked swiftly to create a customized treatment for him using a new gene-editing technique called CRISPR.
When he was seven months old, the treatment was first administered, and it appears to have been effective. KJ rapidly gained weight that was suitable for a baby of his age as his body, which was battling a toxic buildup of ammonia, started to flourish.
After 307 days at Children’s Hospital of Philadelphia, baby KJ was released home to his parents and siblings on Tuesday, wearing a cap and gown to represent his “graduation” from the hospital. He was escorted home by local law enforcement after doctors and nurses gathered for a “clap out” as he left the hospital.
Approximately 1 in 1 to 3 million people have KJ’s metabolic disorder, carbamoyl-phosphate synthetase 1 deficiency. By early infancy, half of the infants die from the disease.
At Children’s Hospital of Philadelphia, Dr. Dot Ahrens-Nicklas, one of KJ’s physicians, told ABC News that KJ “had the most severe variant.”.
“We had to accelerate the personalized therapy pathway we were already working on,” he stated.
There are already approved gene therapy treatments for more prevalent genetic diseases, such as beta thalassemia and sickle cell disease, which affect tens of thousands of people in the United States. S. Prominent biotechnology and pharmaceutical companies sell those treatments.
Due to the rarity of KJ’s illness, his doctors had to work alone. But because of the technology at Children’s Hospital of Philadelphia, his doctors thought they could create their own customized therapy in-house using a more efficient method.
For a rare genetic condition like KJ’s and possibly for future children born with slightly different genetic errors, CRISPR gene-editing technology was the ideal tool.
According to Dr. Kiran Musunuru, director of the Genetic and Epigenetic Origins of Disease Program at the Penn Cardiovascular Institute, “think of it like a GPS signal,” ABC News reported. Depending on the precise gene sequence you wish to alter, you can alter the GPS’s direction. “.
Musunuru is optimistic that more babies with extremely uncommon conditions will be able to receive this type of treatment, but he acknowledges that much more work needs to be done to make it possible.
In this report, Dr. Keerthana Pakanati of ABC contributed.
