Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder

MedicalXpress | Apr 14, 2026

In 2025, baby KJ Muldoon became the first person to receive a personalized gene editing treatment, which likely saved his life. But the scientific advances that made the groundbreaking treatment possible were years in the making long before KJ was born. One was base editing, the technology developed in 2016 by David Liu and his lab at the Broad Institute that makes single-letter changes in DNA and was used to correct KJ's life-threatening mutation.