Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease - Kiran Musunuru; Sarah A. Grandinette; Xiao Wang; Taylor R. Hudson; Kevin Briseno; Anne Marie Berry; Julia L. Hacker; Alvin Hsu; Rachel A. Silverstein; Logan T. Hille; Aysel N. Ogul; Nancy A. Robinson-Garvin; et al. (New England Journal of Medicine, 2025)