We evaluated transcriptional changes elicited by targeting AAVS1 and IL2RG genes and we found that the impact of one or few nuclease-induced DNA DSBs is minor and mainly limited to genes belonging to
We performed single-cell (sc)RNA-Seq on male donors FACS-sorted âprimitiveâ (CD34+ CD133+ CD90+) and âprogenitorâ (CD34+ CD133+ CD90-) HSPC cells treated with IL2RG ZFN, High Specificity (HS)
