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Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy

Proximal spinal muscular atrophy (SMA), one of the most common genetic causes of infant death, results from the selective loss of motor neurons in the spinal cord. SMA is a consequence of low levels of survival motor neuron (SMN) protein. In humans, the SMN gene is duplicated; SMA results from the l...

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Detalhes bibliográficos
Main Authors: Butchbach, Matthew E.R., Singh, Jasbir, Þorsteinsdóttir, Margrét, Saieva, Luciano, Slominski, Elzbieta, Thurmond, John, Andrésson, Thorkell, Zhang, Jun, Edwards, Jonathan D., Simard, Louise R., Pellizzoni, Livio, Jarecki, Jill, Burghes, Arthur H.M., Gurney, Mark E.
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2010
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2798721/
https://ncbi.nlm.nih.gov/pubmed/19897588
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddp510
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