Development of a Protein-Based Therapy for the Treatment of Spinal Muscular Atrophy
The autosomal recessive disorder spinal muscular atrophy (SMA) causes motor neuron degeneration and muscle wasting, progressing to paralysis and death in severe cases. The disease is caused by deficiency of survival motor neuron protein (SMN) due to deletion or mutation of the SMN1 gene. We seek to...
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| Language: | en |
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2014
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| Online Access: | http://hdl.handle.net/10393/30697 |