Pervasive donor DNA integration defies precision gene editing of hematopoietic stem cells.

Bauer, Daniel E · Cell Stem Cell · 2022

editorial · Level V

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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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