Fig. 3: NT-3 gene transfer improves sciatic nerve histopathology in Cx32 KO mice.
From: AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1
One-micron-thick, toluidine blue-stained cross-sections of sciatic nerves from untreated and NT-3-treated mice (a–e) at 6 months post gene transfer. The thinly myelinated axons commonly seen in the untreated nerves (a) are decreased with treatment (b). Arrows indicate Wallerian degeneration (a), Schmidt–Lanterman incisures (c) onion bulbs (d), and acutely demyelinated axons (e). Efficacy of NT-3 gene therapy assessed by quantification of histopathological findings on sciatic nerves from NT-3 treated and untreated (UT) Cx32 KO mice and WT controls (f). NT-3 treated mice showed improvements in the frequency of Schmidt–Lanterman incisures (SLI), demyelinated axons (DA), onion bulb formations (OBF), and acute Wallerian degeneration (WD). Increase in the number of SLIs and reduction in OBF and WD profiles were observed predominantly in the females (g). Error bars are ±SEM; n = 8 per group, *p < 0.05; **p < 0.01; ***p < 0.001; One-way ANOVA with Tukey’s multiple comparisons test for SLI and unpaired t test for other findings.
