Researchers successfully used a personalized CRISPR-based gene therapy to treat a baby, KJ, with a life-threatening metabolic disorder, CPS1 deficiency. The treatment, detailed in the New England Journal of Medicine, involved editing a specific gene in KJ's liver cells using lipid nanoparticles. Three months post-treatment, KJ shows significant improvement, tolerating higher protein intake and needing less supplemental treatment. While long-term effects need further observation, this groundbreaking approach offers hope for treating various ultra-rare genetic diseases, marking a potential new era of personalized medicine.

Prepared by Olivia Bennett and reviewed by editorial team.