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. Author manuscript; available in PMC: 2022 Feb 1.
Published in final edited form as: Transfus Apher Sci. 2021 Jan 10;60(1):103060. doi: 10.1016/j.transci.2021.103060

Fig. 1. Genome editing based strategy for treating sickle cell disease.

Fig. 1.

CD34+ HSPCs are first isolated from a patient with sickle cell disease. The RNP (ribonecleoprotein) complex of CRISPR guide RNA with Cas9 protein and DNA donor template are delivered into the nuclei of HSPCs via electroporation for gene correction. The gene-edited HSPCs are then infused back into the patient to reverse the disease phenotype. To make the gene-editing strategy a clinically viable approach, both high efficacy and adequate safety need to be achieved.