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. 2020 Jul 29;11:3778. doi: 10.1038/s41467-020-17552-3

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© The Author(s) 2020

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.

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Fig. 3 — a AAV6 donor used for KI experiments of FIX Padua. b FIX KI efficiency in HUDEP-2 cells was measured by flow cytometry (light green) or ddPCR specific for on-target integration (dark green) before and after sorting (n = 1). c Quantification of FIX mRNA in KI HUDEP-2 upon differentiation (mean ± SD, n = 2 undifferentiated, n = 3 differentiated). d Quantification of FIX secretion in medium of HUDEP-2 clones (n = 28) with monoallelic or biallelic KI (ELISA), as detected by on target ddPCR analysis (AAV-genome junction amplification). Lines represent median. e KI efficiency in HSPCs at day 9 of erythroid differentiation. Lines represent mean (n = 4). f, g FIX expression during HSPC differentiation at RNA (f, qPCR; n = 2 day 9; n = 4 day 12) and protein level (g, ELISA on supernatants, n = 3 day 7; n = 4 day 9 and 12; 3 donors). Bars represent mean ± SD. h Comparison of FIX antigen (ELISA) and activity (aPTT) in supernatants of KI HSPCs (mean; n = 2). i, j Comparison of FIX RNA at day 9 and 12 of erythroid differentiation (i) and protein (j) in KI HSPCs (AAV + RNP) vs HSPCs transduced with an erythroid-specific lentiviral vector (LvEry FIX). Bars represent mean (**p = 0.003 t-test Holm-Sidak correction for RNA at day 12; p = 0.08 for protein, n = 2). k Integration pattern in single BFU-E (2 donors): no integration (0), monoallelic (1) and biallelic KI (2). Source data are in the Source Data file.