A Baby Received a Custom Crispr Treatment in Record Time

Last August, KJ Muldoon was born with a potentially fatal genetic disorder. Just six months later, he received a Crispr treatment designed just for him. Muldoon has a rare disorder known as CPS1 deficiency, which causes a dangerous amount of ammonia to build up in the blood. About half of babies born with it will die early in life. Current treatment options—a highly restrictive diet and liver transplantation—aren’t ideal. But a team at the Children’s Hospital of Philadelphia and Penn Medicine was able to bypass the standard years-long drug development timeline and use Cripsr to create a personalized medicine for KJ in a matter of months. “We had a patient who was facing a very, very devastating outcome,” says Kiran Musunuru, professor for translational research at the University of Pennsylvania and Children’s Hospital of Philadelphia, who was part of the team that made KJ’s treatment. When KJ was born, his muscles were rigid, he was lethargic, and he wouldn’t eat. After three doses ... Read full article.