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Decreasing Disease Severity in Symptomatic, Smn(−/−);SMN2(+/+), Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery
Spinal muscular atrophy (SMA), an autosomal recessive neuromuscular disorder, is the leading genetic cause of infant mortality. SMA is caused by the homozygous loss of Survival Motor Neuron-1 (SMN1). In humans, a nearly identical copy gene is present, SMN2. SMN2 is retained in all SMA patients and e...
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| Main Authors: | , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Mary Ann Liebert, Inc.
2012
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3300100/ https://ncbi.nlm.nih.gov/pubmed/22029744 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.166 |
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