Groundbreaking Personalized Gene Therapy Saves Infant with Rare, Incurable Disease
Joshua Fu2025-05-20T21:27:55+00:00An NIH-supported team has delivered the first personalized CRISPR-based gene therapy to an infant with CPS1 deficiency, offering new hope for rare disease treatment. The customized approach shows early success and may pave the way for future individualized therapies...