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

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.

Automated summary

In this study, Ferrari et al. identified issues with commonly used AAV donors in gene editing, including weakened hematopoietic repopulation and frequent genomic integrations.