Engraftment and persistence of HBB base-edited hematopoietic stem cells in nonhuman primates
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Sickle cell disease (SCD) is caused by a single nucleotide change in the β-globin gene that adenine base editors can convert to the nonpathogenic Makassar β-globin variant. Here, we evaluated the long-term efficiency and off-target editing potential of autol... ...
