Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by selective loss of motor neurons. Vascular endothelial growth factor (VEGF) is a key mediator of angiogenesis, and has neurotrophic and neuroprotective activities. To examine the efficacy of VEGF electro-gene therapy for ALS, intramuscular mouse VEGF 164 gene transfer was performed by electroporation to treat a mouse model of ALS, transgenic Cu/Zn superoxide dismutase (SOD1) (G93A) mice. VEGF electro-gene therapy delayed the onset of decline in hindlimb function by more than 1 week, but did not significantly prolong survival in SOD1 transgenic mice, suggesting that continuous release of VEGF 164 by intramuscular electro-gene therapy is partially effective in the treatment of SOD1 transgenic mice.

Keywords: Amyotrophic lateral sclerosis, electroporation, gene therapy, grip strength, intramuscular injection, motor neuron, neuromuscular junction, rotarod test, transgenic SOD1 G93A mice, vascular endothelial growth factor.