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A Baby Received a Custom Crispr Treatment in Record Time
Scientists were able to create a bespoke treatment for KJ Muldoon’s rare genetic disorder within six months. It could be a blueprint for potentially life-saving, gene-editing Crispr therapies.
“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. The case is detailed today in a study published in The New England Journal of Medicine and was presented at the American Society of Gene & Cell Therapy annual meeting in New Orleans. They decided to focus on urea cycle disorders, a group of genetic metabolic conditions that affect the body’s ability to process ammonia that includes CPS1 deficiency.
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