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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

2021Nature380 citationsDOIOpen Access PDF

Topics & Concepts

HaematopoiesisStem cellBone marrowProgenitor cellTransplantationBiologyGenome editingGenetic enhancementGlobinImmunologyCancer researchMedicineGeneCell biologyGeneticsCRISPRInternal medicineCRISPR and Genetic EngineeringRNA regulation and diseaseHemoglobinopathies and Related Disorders
Base editing of haematopoietic stem cells rescues sickle cell disease in mice | Litcius