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Since its advent in 2012, CRISPR genome-editing technology has held the potential to change medicine. But getting from a research breakthrough to practical impacts on patients is no simple feat. In 2025, doctors Kiran Musunuru and Rebecca Ahrens-Nicklas showed it can be done. When baby KJ was born with a rare metabolic disease, giving him a 50% chance of dying in infancy, the pair led a remarkable multi-institutional team that designed, tested, and administered a personalized gene-editing therapy in just six months. Baby KJ is now home with his family and recently took his first steps.
