Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells

Abstract excerpt

Abstract Some gene polymorphisms can lead to monogenic diseases, whereas other polymorphisms may confer beneficial traits. A well-characterized example is congenital erythrocytosis—the non-pathogenic hyper-production of red blood cells—that is caused by a truncated erythropoietin receptor. Here we show that Cas9-mediated genome editing in CD34 + human haematopoietic stem and progenitor cells (HSPCs) can recreate...

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