Article
Prime editing in hematopoietic stem cells—From ex vivo to in vivo CRISPR-based treatment of blood disorders
10 Mar 2023
Abstract excerpt
Prime editing of human hematopoietic stem cells has the potential to become a safe and efficient way of treating diseases of the blood directly in patients. By allowing site-targeted gene intervention without homology-directed repair donor templates and DNA double-stranded breaks, the invention of prime editing fuels the exploration of alternatives to conventional recombination-based ex vivo genome editing of...
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