Gene replacement therapy in a model of Charcot-Marie-Tooth 4C neuropathy.

Myelin thickness of all size fibres was increased in treated mice (1.234 ± 0.407 μm) compared to the mock group (0.899 ± 0.311 μm), an increase that did not reach statistical significance ( P > 0.05).

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This analysis showed a trend for ameliorated overexpression of dysregulated genes in both lumbar roots and sciatic nerves from treated mice, including Pou3f1 /Scip, Cdgap , and Cxcl14 , but only Pou3f1 /Scip downregulation in the sciatic nerves was statistically significant ( Supplementary Fig. 10 B and C).

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