Figure 3.

Ex vivo vs. in vivo HSC gene-editing. Steps shown for Ex vivo editing are widely applied for gene editing in in vivo animal models and now also in clinical trials for gene editing. Findings for therapy by gene addition indicate that gene editing, too, might benefit from selective HSC depletion by delivery of antibody-drug conjugates [55] and for suitable disorders, such as FA, from engraftment of corrected cells without conditioning [14]. Steps shown for In vivo editing are in part extrapolated for HSC-targeted approaches of gene addition [56,57,58] and in part based on the latest developments and concepts in the delivery of gene editing components and mRNAs [59,60,61,62].