Scientists utilized CRISPR technology to develop an innovative drug for KJ Muldoon, a newborn suffering from CPS1 deficiency. This rare condition, impacting the liver's ability to process protein byproducts, can lead to lethal ammonia buildup in the blood. KJ's situation was dire, and his medical team proposed a novel approach, creating a completely tailored treatment to address his genetic mutation. With overwhelming dedication from researchers nationwide, the experimental therapy succeeded, marking a transformative moment in genetic medicine and hope for patients with similar conditions.
"We had a patient who was facing a very, very devastating outcome," Kiran Musunuru, a professor at the University of Pennsylvania and CHOP who was on the team that treated KJ.
"We loved him," Nicole Muldoon, KJ's mother, told The New York Times, "and we didn't want him to be suffering."
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