Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegia
J. Clin. Invest. Marinella Pirozzi, et al. 116:202 doi:10.1172/JCI26210 [Go to this article.]

Figure 5
Rescue of the ultrastructural mitochondrial abnormalities. (A–G) Electron micrographs of the sciatic nerves in 16-month-old Spg7^–/– mice. Mice were analyzed 6 months after intramuscular injection with AAV-LacZ on the left side (A, C, and F) and AAV2/2-Spg7 on the right side (B, D, E, and G). A significant reduction in the number of axons containing abnormal mitochondria is observed in the sciatic nerve from the side injected with the AAV2/2-Spg7 vector (B). Giant mitochondria with disrupted cristae and glycogen accumulation (asterisk in F) are detected in the axons from the side treated with the control vector (C and F), while normal mitochondria with well-preserved cristae are present on the contralateral side (D, E, and G). (H) Morphometric quantification of the percentage of sciatic nerve axons with abnormal mitochondria. Upon injection of AAV2/2-Spg7, the number of axons with abnormal mitochondria significantly decreases compared with the contralateral side and drops below the levels that were present at the time of injection. The P values of Student’s t tests are shown. Scale bar: 5 μm (A and B); 1.5 μm (C, D, and F); 1 μm (E); 400 nm (G).