Intracerebroventricular SPAST-AAV9 gene therapy prevents manifestation of symptoms in a mouse model of SPG4 hereditary spastic paraplegia.

Abstract excerpt

Hereditary spastic paraplegia type 4 is characterized by gait impairments, progressive spasticity, and weakness of the lower limbs, resulting from degeneration of the corticospinal tracts. The disease is caused by mutations of the SPAST gene, which encodes a major isoform of spastin called M87 and a minor isoform called M1. Owing to its N-terminal hydrophobic domain not shared by M87, M1 is the isoform that...

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• Dependovirus
• Disease Models, Animal
• Gene Expression
• Genetic Therapy
• Genetic Vectors