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Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?
Nat Rev Neurosci. 2009 Aug;10(8):597-609. doi: 10.1038/nrn2670. Epub 2009 Jul 8.
Nat Rev Neurosci. 2009.
PMID: 19584893
Free PMC article.
Review.
Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect.
Gavrilina TO, McGovern VL, Workman E, Crawford TO, Gogliotti RG, DiDonato CJ, Monani UR, Morris GE, Burghes AH.
Gavrilina TO, et al.
Hum Mol Genet. 2008 Apr 15;17(8):1063-75. doi: 10.1093/hmg/ddm379. Epub 2008 Jan 4.
Hum Mol Genet. 2008.
PMID: 18178576
Free PMC article.
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