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. Author manuscript; available in PMC: 2020 Jun 5.
Published in final edited form as: Nat Med. 2019 Oct 7;25(10):1505–1511. doi: 10.1038/s41591-019-0594-0

Extended Data Fig. 7 |. Homogeneously expressed recombinant μutro normalizes β-sarcoglycan, reverses myopathology and normalizes fiber size in GRMD dogs after systemic gene delivery.

Extended Data Fig. 7 |

a, Immunofluorescence staining detecting the utrophin N terminus (Utro N, red) and β-sarcoglycan (green) of vastus lateralis muscle from wild type (WT), treated (AAV-μUtro) or untreated (PBS). The experiment was repeated independently at least two times with similar results. b, H&E staining of vastus lateralis muscle biopsies. Scale bar, 100 μm c, Distribution of minimum Feret diameter of myofibers in vastus lateralis muscle biopsies pooled from age-matched wild type (n = 920) 19 ± 4.01, GRMD littermates randomized to AAV-μUtro (n = 758) 17.63 ± 4.9, and PBS (n = 1,014) 14.49 ± 7.24. Data are presented as mean ± s.d. Coefficients of variation for all groups are reported in the box. d, Cumulative distribution (percentage) plot of minimum Feret diameter from c. e, Distribution of minimum Feret diameter of myofibers in vastus lateralis muscle biopsies obtained at necropsy, wild type (n = 569) 28.26 ± 8.39, AAV-μUtro (n = 542) 28.4 ± 7.54 and PBS (n = 317) 32.26 ± 12.01. Data are presented as mean ± s.d. Coefficients of variation for all groups are reported in the box. f, Cumulative distribution (percentage) plot of minimum Feret diameter from e. Coefficients of variation for all groups are reported in the box. (See Source Data Extended Data Fig. 7).