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 [Go to this article.]

Figure 3
Retrograde lentivector delivery of SMN at the onset of disease extends survival and delays the phenotype in SMA mice. (A) Weight measurements of animals treated with lentivector-SMN or lentivector-LacZ. (B) Survival analysis in SMA mice injected at 2 days of age with lentivector-LacZ or lentivector-SMN. Mortality was significantly delayed in mice treated with lentivector-SMN compared with the control LacZ group. (C) Image showing lentivector-mediated expression of SMN in spinal MN at the end stage of disease as monitored using Abs against the HA tag. Arrow indicates gems. (D) Double labeling using HA (green, arrow indicates gems) and CGRP Abs (red) in spinal sections. No HA staining was detected in LacZ-injected mice (E). (F and G) SMN expression in muscles from SMN and LacZ-treated mice, respectively. Arrows in F indicate lentivector-mediated SMN expression. (H) Western blot analysis of ventral spinal cord in LacZ, SMN, and WT animals. Scale bars: 50 μm (C–E), 100 μm (F and G).