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Homology-directed gene-editing approaches for hematopoietic stem and progenitor cell gene therapy

9 Sept 2021

Abstract excerpt

The advent of next-generation genome engineering tools like CRISPR-Cas9 has transformed the field of gene therapy, rendering targeted treatment for several incurable diseases. Hematopoietic stem and progenitor cells (HSPCs) continue to be the ideal target cells for gene manipulation due to their long-term repopulation potential. Among the gene manipulation strategies such as lentiviral gene augmentation,...

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PMID: 34503562
DOI: 10.1186/s13287-021-02565-6

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