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 1EIAV-based lentivector-mediated gene transfer in vivo. Transverse sections of lumbar spinal cord (
A), brain stem (
B and
C), and muscle (
D) showing transduction of both muscle and MNs after injection of 30 μl rabies G pseudotyped lentivector-LacZ vector in the gastrocnemius (
A) and facial muscles (
B and
C) of postnatal P2 FVB mice. (
E) Cell counts of total transduced MNs in the lumbar spinal cord, facial nucleus, and motor trigeminal nucleus (MTN) after intramuscular injections of lentivector-LacZ into 2-day-old SMA mice. Data are means ± SEM. Expression of β-gal (green) (
F) colocalizes with the immunofluorescence of CGRP (red) in spinal MNs, producing yellow staining (
G). Scale bars: 400 μm (
A and
B), 200 μm (
C and
D), 100 μm (
F and
G).
