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 3
Paraplegin expression in skeletal muscle and spinal cord mitochondria after AAV-mediated intramuscular delivery of Spg7 cDNA. Western blot analysis of paraplegin on mitochondrial extracts from the gastrocnemius and the spinal cord of Spg7^–/– mice injected intramuscularly unilaterally with AAV2/1-Spg7 (A) and AAV2/2-Spg7 (B). In both cases, the contralateral side was injected with AAV-LacZ. For control we loaded mitochondrial extracts deriving from WT mice. With both serotypes, paraplegin is absent in muscles injected with AAV-LacZ but is restored upon injection of AAV-Spg7. The level of transduction in the muscle is higher when using AAV2/1, as expected. In the spinal cord, paraplegin is detected only when using AAV2/2-Spg7.