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

@inproceedings{Dirren2015SOD1SI,
  title={SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice},
  author={Elisabeth Dirren and Julianne Aebischer and Cylia Rochat and Christopher L Towne and Bernard L Schneider and P. Aebischer},
  booktitle={Annals of clinical and translational neurology},
  year={2015}
}
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… CONTINUE READING
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