Exploring Novel Methods to Achieve Systemic Delivery of SMN for Treatment of Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an inherited neurodegenerative disease caused by insufficient levels of the survival motor neuron protein (SMN), leading to progressive deterioration of α-motor neurons, onset of muscle atrophy and, in severe disease, death. We investigated whether reducing the size...
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| Language: | en |
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Université d'Ottawa / University of Ottawa
2014
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| Online Access: | http://hdl.handle.net/10393/31803 http://dx.doi.org/10.20381/ruor-6563 |