Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy
J. Clin. Invest. Mimoun Azzouz, et al. 114:1726 doi:10.1172/JCI22922 [
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Figure 4SMN gene replacement protects spinal and brain stem MNs in SMA mice. ChAT immunoreactivity in lentivector-SMN–treated (
A, insert) and lentivector–Lac-Z–treated (
B, insert) mice. Immunohistochemistry showing CGRP-positive MNs in lumbar spinal cord of lentivector-SMN–injected (
C) and lentivector-LacZ–injected (
D) animals. (
E) Cell counts of surviving lumbar spinal cord MNs in WT (control), lentivector–Lac-Z–, and lentivector-SMN–treated SMA mice at end stage of disease. (
F) Quantification of facial nucleus MNs in control, lentivector-LacZ–, and lentivector-SMN–injected animals at end stage of disease. Scale bars: 100 μm.