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. 2017 Aug 25;7:9473. doi: 10.1038/s41598-017-09958-9

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© The Author(s) 2017

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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Neonatal UCBT prevents GAGs accumulation in MPS-I mice. (A) Donor chimerism (percentage of CD45.1⁺ cells) determined by flow cytometry in the PB of recipient MPS-I and WT mice at 20 weeks (time of sacrifice) after nUCBT (n = 12 for MPS-I, n = 12 for WT; p = 0.24 by Wilcoxon test). Dashed line indicates the level of 50% donor engraftment, and identifies the highly-engrafted mice group (with ≥50% donor cells in PB, nUCBT-hi). (B) IDUA activity in spleen, liver, lung, kidney, and heart of WT (n = 8), MPS-I (n = 8), MPS-I nUCBT-hi (n = 5), and MPS-I nUCBT-lo mice (n = 5). (C) GAG levels in the indicated organs of the same WT, MPS-I, MPS-I nUCBT-hi, and MPS-I nUCBT-lo mice. (D) Levels of ΔDiHS-0S, ΔDiHS-NS, ΔDi-4S, and mono-sulfated KS in the plasma of the mice. *p ≤ 0.05, **p ≤ 0.01, ***p ≤ 0.001 by Wilcoxon test.