In a World First, CRISPR Drug Tailored for One Baby Shows Life-Saving Promise

Image by Getty / Futurism Treatments Scientists used CRISPR tech to create a custom-made drug targeted at helping a newborn baby fight a rare genetic disorder. According to an extraordinary new paper published in The New England Journal of Medicine, the drug worked — marking the first time that a patient was successfully healed with a bespoke gene-hacked medicine, and a possible watershed moment for the field of genetic medicine. The baby, named KJ Muldoon, was born at the Children's Hospital of Philadelphia (CHOP) and at Penn Medicine, with a rare genetic disorder called CPS1 deficiency. According to the National Institutes of Health (NIH), the condition occurs when a person's liver cells are unable to break down protein byproducts, causing dangerous levels of ammonia to build up in their blood. It's painful, and often deadly; about half of children diagnosed with the disorder die in early infancy. Patients who live are limited to extremely strict diets until they're old enough to — ... Read full article.