SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice.

ABSTRACT:

Objective

Amyotrophic lateral sclerosis is an incurable disorder mainly characterized by motoneuron degeneration. Mutations in the superoxide dismutase 1 (SOD1) gene account for 20% of familial forms of the disease. Mutant SOD1 exerts multiple pathogenic effects through the gain of toxic properties in both neurons and glial cells. Here, we compare AAV-based gene therapy suppressing expression of mutant SOD1 in either motoneurons or astrocytes.

Methods

AAV vectors encoding microRNA against human SOD1 were administered to (G93) (A)SOD1 mice either by intracerebroventricular injections in pups or by lumbar intrathecal injections in adults. Vector systems were designed to suppress SOD1 expression predominantly in either spinal motoneurons or astrocytes. Electrophysiological and

SUBMITTER: Dirren E 

PROVIDER: S-EPMC4338957 | biostudies-literature | 2015 Feb

REPOSITORIES: biostudies-literature