In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice
Luke W. Koblan, Michael R. Erdos, Christopher Wilson, Wayne A. Cabral, Jonathan M. Levy, Zheng‐Mei Xiong, Urraca Tavarez, Lindsay M. Davison, Yantenew Gete, Xiaojing Mao, Gregory A. Newby, Sean Doherty, Narisu Narisu, Quanhu Sheng, Chad Krilow, Charles Y. Lin, Leslie B. Gordon, Kan Cao, Francis S. Collins, Jonathan D. Brown, David R. Liu
Topics & Concepts
ProgeriaLMNALaminBiologyRNA splicingMutationRNAMolecular biologyTransgeneGeneticsPremature agingGeneNuclear Structure and FunctionRNA regulation and diseaseCRISPR and Genetic Engineering