Figure - PMC

Skip to main content

An official website of the United States government

Here's how you know

Here's how you know

Official websites use .gov
A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS
A lock ( ) or https:// means you've safely connected to the .gov website. Share sensitive information only on official, secure websites.

View full-text article in PMC

. 2024 Jun 11;15:4965. doi: 10.1038/s41467-024-49353-3

Search in PMC
Search in PubMed
View in NLM Catalog
Add to search

© The Author(s) 2024

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 licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence 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 licence, visit http://creativecommons.org/licenses/by/4.0/.

PMC Copyright notice

Fig. 4 — a Representative schema of gene correction assessment in HSPCs. Frequency of homology-directed repair (HDR) and insertion/deletion (indels) allelic events (b) or ratio HDR/indel (c) at HBB locus measured at day 4 by ddPCR in PLX HSPCs edited with TALEN and ssODN or AAV in presence (+) or absence (−) of HDR-Enh01 and Via-Enh01. Data are expressed as average ± SD for n = 4 (−, ssODN), n = 7 (−, AAV), n = 6 (+, ssODN) and n = 9 (+, AAV) biologically independent experiments and donors. Kruskal-Wallis followed by Dunn’s multi-comparison test. P-values are indicated. d Frequency of viable cells and CD34-expressing cells evaluated at day 4 in HSPCs edited by ssODN optimized (n = 6) and non-optimized (n = 4) protocols or by AAV optimized (n = 10) and non-optimized (n = 7) protocols compared to untreated cells (n = 15). e CFU plating efficiency obtained for HSPCs edited by ssODN optimized (n = 5) and non-optimized (n = 3) protocols or by AAV optimized (n = 6) and non-optimized (n = 6) protocol compared to untreated cells (n = 12). On each box plot, the central mark indicates the median plating efficiency, the bottom and top edges of the box indicate the interquartile range (IQR), and the whiskers represent the maximum and minimum data point. Each point represents one experiment performed with a given donor. One-way ANOVA with Tukey’s comparison test. P-values are indicated. f Average frequency of CFU-GM, BFU-E and CFU-GEMM colonies ± SD obtained for untreated HPSCs (n = 12) or HSPCs edited by ssODN optimized (n = 5) or non-optimized (n = 3) protocols or AAV optimized (n = 6) or non-optimized (n = 6) protocols. One-way ANOVA with Tukey’s comparison test. P-value are documented in the Source data file. g Frequency of allelic editing events detected in single BFU-E colonies enumerating bi-allelic genotype as β = corrected allele, β^s = sickle allele_, β⁰=indels allele. Data represent editing events detected in ssODN-edited groups (optimized and non-optimized protocols, n = 6 and n = 4 independent biological experiments and donors, respectively) and in AAV-edited groups (optimized and non-optimized protocols, n = 6 independent biological experiments and donors in both conditions). One-way ANOVA with Tukey’s comparison test. P-values are indicated. Source data are provided as a Source data file.