Baby Receives First Ever Gene Editing Treatment To Treat Rare Disorder
This is truly remarkable and could transform healthcare as we know it! More than 30 million people in the United States are affected with any of some 7000 genetic disorders.
Kyle and Nicole Mudoon’s baby was one of those, born with a rare genetic disorder that only impacts one in 1.3 million babies. CPS1 deficiency causes severe mental and developmental delays, and often results in death. As Mudoon recalled doctors telling them, “We either have to get him a liver transplant, or give him a treatment that’s never been given to anybody before, right? I mean, what an impossible decision to make?” They decided the best option would be to try this gene-editing treatment designed just for KJ. The treatment finds the one letter that needs to be changed out of the 3 billion in your body.
KJ received three infusions and tolerated them so well, he started hitting milestones. He’s now 40th percentile in weight compared to his previous 7th, and is getting ready to be released from the hospital for the first time. He’s 9 and 1/2 months old.
KJ’s treatment — which was built on decades of research and by scientists who put all other work aside for 6 months — offers a new path for companies to develop personalized treatments without going through years of expensive development and testing. It eventually could also be used for more common genetic disorders like sickle cell disease, cystic fibrosis, Huntington’s disease and muscular dystrophy.