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Therapy Development by Genome Editing of Hematopoietic Stem Cells

14 Jun 2021

Abstract excerpt

Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the blood and immune systems has placed them at the forefront of cell and gene therapy development. Recent advances in genome-editing tools, in particular for clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) and CRISPR/Cas-derived editing systems, have transformed the gene...

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PMID: 34198536
DOI: 10.3390/cells10061492

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