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. 2018 Aug 14;10:313–326. doi: 10.1016/j.omtm.2018.08.003

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© 2018 The Author(s)

This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

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Expansion of Normal and Thalassemic Cells before or after Editing

(A) Flow chart describing the expansion after transfection protocol. Cells sorted on day 1 (without expansion) were used as a control. (B) Flow chart describing the transfection after expansion protocol. (C) Indel levels in different sorted progenitor and stem cell populations from the three editing protocols. (D) Fold increase of the total numbers of CD34+/CD38−/CD90+ edited cells. (E) Human chimerism of unexpanded and SR1+Ly (SL) expanded enhancer-edited normal cells. (F) Indel percentage in the peripheral blood of primary recipients (top) and estimation of the percentage of edited cells in peripheral blood (bottom). Top left: the frequency of edited total input cells. *p < 0.001 versus unexpanded.