Using the BTX ECM 830 Electroporator to Enable CRISPR/HDR Delivery in Human Hematopoietic Stem Cells
In this study, the BTX ECM 830 electroporator was used to deliver CRISPR-Cas9 ribonucleoprotein (RNP) complexes and donor DNA templates into human CD34+ hematopoietic stem and progenitor cells (HSPCs) via electroporation.
This study compared the long-term effects of CRISPR-Cas9 with homology-directed repair (HDR) and lentiviral transduction on human hematopoietic stem cells (HSCs). While both methods can successfully modify CD34+ HSCs, only lentiviral-edited cells maintained robust long-term engraftment, clonal diversity, and multilineage differentiation in immunodeficient mice. In contrast, CRISPR/HDR-edited cells showed reduced engraftment, limited clonal representation, and signs of altered gene expression that may impair stem cell function. These findings highlight a trade-off between the precision of CRISPR editing and the durability of HSCs, with important implications for gene therapy applications.
In this study, the BTX ECM 830 electroporator was used to deliver CRISPR-Cas9 ribonucleoprotein (RNP) complexes and donor DNA templates into human CD34+ hematopoietic stem and progenitor cells (HSPCs) via electroporation. This technique allowed the researchers to perform precise gene editing using homology-directed repair (HDR) at the AAVS1 safe harbor locus.
A link to the article can be found here.
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