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Updated: Aug 26, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
Pervasive donor DNA integration defies precision gene editing of hematopoietic stem cells
1Division of Hematology/Oncology, Boston, MA 02115, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA 02115, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA; Broad Institute, Cambridge, MA 02142, USA; Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA.
Abstract:
Therapeutic gene editing based on homology-directed repair (HDR) promises to precisely recombine donor template sequences at programmed double-strand break sites. In this issue, Ferrari et al. report that commonly used AAV donors blunt hematopoietic repopulation and result in frequent genomic integrations that could interfere with normal gene expression.
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