Base editing of haematopoietic stem cells rescues sickle cell disease in mice
Gregory A. Newby, Jonathan Yen, Kaitly J. Woodard, Thiyagaraj Mayuranathan, Cícera R. Lazzarotto, Yichao Li, Heather Sheppard, Shaina N. Porter, Yu Yao, Kalin Mayberry, Kelcee A. Everette, Yoonjeong Jang, Christopher J. Podracky, Elizabeth Thaman, Christophe Lechauve, Akshay Sharma, Jordana M. Henderson, Michelle F. Richter, Kevin T. Zhao, Shannon M. Miller, Tina Wang, Luke W. Koblan, Anton P. McCaffrey, John F. Tisdale, Theodosia A. Kalfa, Shondra M. Pruett‐Miller, Shengdar Q. Tsai, Mitchell J. Weiss, David R. Liu
Topics & Concepts
HaematopoiesisStem cellBone marrowProgenitor cellTransplantationBiologyGenome editingGenetic enhancementGlobinImmunologyCancer researchMedicineGeneCell biologyGeneticsCRISPRInternal medicineCRISPR and Genetic EngineeringRNA regulation and diseaseHemoglobinopathies and Related Disorders