ALS Compendium

Title: Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic benefit in fALS mice

Year Published: 2011

Observation: AAV:shSOD1tranduces MNs after neonatal intramuscular delivery and silences mSOD1 in vivo. There was near complete protection from MN loss at 110 days. mSOD1 silencing doesn't alter activation of glial cells. Multiple injections of AAV:shSOD1 doesn't improve motor performance or increase survival

Comment: No effect on motor performance or survival after multiple injections of AAV:shSOD1

Citation: Towne, C., Setola, V., Schneider, B. L., & Aebischer, P. (2011). Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic benefit in fALS mice. Molecular Therapy : The Journal of the American Society of Gene Therapy, 19(2), 274–83. doi:10.1038/mt.2010.260

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