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Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect.
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.
A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy.
Monani UR, Pastore MT, Gavrilina TO, Jablonka S, Le TT, Andreassi C, DiCocco JM, Lorson C, Androphy EJ, Sendtner M, Podell M, Burghes AH.
Monani UR, et al. Among authors: gavrilina to.
J Cell Biol. 2003 Jan 6;160(1):41-52. doi: 10.1083/jcb.200208079. Epub 2003 Jan 6.
J Cell Biol. 2003.
PMID: 12515823
Free PMC article.
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SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN.
Le TT, Pham LT, Butchbach ME, Zhang HL, Monani UR, Coovert DD, Gavrilina TO, Xing L, Bassell GJ, Burghes AH.
Le TT, et al. Among authors: gavrilina to.
Hum Mol Genet. 2005 Mar 15;14(6):845-57. doi: 10.1093/hmg/ddi078. Epub 2005 Feb 9.
Hum Mol Genet. 2005.
PMID: 15703193
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Embryonic motor axon development in the severe SMA mouse.
McGovern VL, Gavrilina TO, Beattie CE, Burghes AH.
McGovern VL, et al. Among authors: gavrilina to.
Hum Mol Genet. 2008 Sep 15;17(18):2900-9. doi: 10.1093/hmg/ddn189. Epub 2008 Jul 3.
Hum Mol Genet. 2008.
PMID: 18603534
Free PMC article.
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