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In a historic first, doctors used CRISPR-based gene editing to treat a baby with a fatal genetic disorder, marking a breakthrough in personalized medicine. KJ Muldoon, born with CPS1 deficiency—a condition that causes toxic ammonia buildup—received a customized therapy developed by researchers at Children’s Hospital of Philadelphia and Penn Medicine. Instead of cutting DNA, the team used a base editing technique to correct a single faulty genetic letter, offering a potential alternative to liver transplant.⁠

KJ began receiving the therapy in February through an IV infusion of lipid nanoparticles targeting liver cells. “This is the first step towards the use of gene editing therapies to treat a wide variety of rare genetic disorders for which there are currently no definitive medical treatments,” said Dr. Kiran Musunuru, co-author of the study published in the New England Journal of Medicine. The project, partly funded by the National Institutes of Health, cost roughly the same as a transplant.⁠

His parents, Kyle and Nicole Muldoon, opted for the experimental treatment after weighing it against surgery. “We prayed, we talked to people, we gathered information, and we eventually decided that this was the way we were going to go,” Kyle said. While researchers stress that long-term outcomes are still unknown, early results have raised hopes the technique could expand to treat other rare genetic diseases.⁠

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In a historic first, doctors used CRISPR-based gene editing to treat a baby with a fatal genetic disorder, marking a breakthrough in personalized medicine. KJ Muldoon, born with CPS1 deficiency—a cond...
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