Gene editing of hematopoietic stem and progenitor cells offers promise as a curative treatment for chronic granulomatous disease (CGD). Here, the authors develop a D10A Cas9n based gene editing strategy to treat CGD with no detectable off-target activity or chromosomal translocations.
- Jonas Holst Wolff
- Thomas Wisbech Skov
- Jacob Giehm Mikkelsen
