Optimized guide RNA selection recommendations for using spCas9 gene editing in human hematopoietic stem and progenitor cells
Han J. M. P. Verhagen, Carlijn Kuijk, Anne M. Kokke, Laurens Rutgers, Santhe A. van der Meulen, Gerard J. van Mierlo, Carlijn Voermans, Emile van den Akker
The paper evaluates sgRNA design for spCas9-RNP gene editing in HSPCs, showing in vitro screening outperforms in silico models and no adverse effects on cell function.
bioRxiv · Dec 18, 2020 · PreprintMolecular Biology