Article
Inhibition of apoptosis blocks human motor neuron cell death in a stem cell model of spinal muscular atrophy.
PloS one - 1 Jan 2012
Sareen Dhruv, Ebert Allison D, Heins Brittany M, McGivern Jered V, Ornelas Loren, Svendsen Clive N
Abstract excerpt
Spinal muscular atrophy (SMA) is a genetic disorder caused by a deletion of the survival motor neuron 1 gene leading to motor neuron loss, muscle atrophy, paralysis, and death. We show here that induced pluripotent stem cell (iPSC) lines generated from two Type I SMA subjects-one produced with lentiviral constructs and the second using a virus-free plasmid-based approach-recapitulate the disease phenotype and...
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