Gene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathy.

In anterior lumbar roots ( Figures 3 C, 3D, 3G, and 3H), the ratio of abnormally myelinated fibers reached 0.25 ± 0.01 (n = 11) in mock compared to 0.15 ± 0.02 in treated mice (n = 11; p = 0.0002; Figure 3 K), while the numbers of foamy macrophages showed a trend for decrease in treated (9.18 ± 1.66; n = 11) compared to the mock-treated mice (13.35 ± 1.21; n = 11; p > 0.05; Figure 3 L).

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