Article

Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells

2026-04-14

Abstract excerpt

First-generation genome editing therapies have largely focused on correcting or compensating for pathogenic variants. However, as these approaches enter the clinic, emerging biological constraints limit maximal therapeutic impact. Because globin genes are activated late during erythroid differentiation, genome-corrected hematopoietic stem and progenitor cells (HSPCs) gain little selective advantage in the bone mar...

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Literature Corpus work: 90846d73-e88c-56ef-a884-1bd782d0fec4
DOI: 10.64898/2026.04.13.718029

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