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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Autori principali: Glascock, Jacqueline J., Osman, Erkan Y., Wetz, Mary J., Krogman, Megan M., Shababi, Monir, Lorson, Christian L.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2012
Soggetti:
Brief Report
Accesso online: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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