Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

By Gavrilina Tatiana O.   McGovern Vicki L.   Workman Eileen   Crawford Thomas O.   Gogliotti Rocky G.   DiDonato Christine J.   Monani Umrao R.   Morris Glenn E.   Burghes Arthur H.M.  

Human Molecular Genetics, Vol. 17, Iss. 8, 2008-04 ,pp. : 1063-1075

Oxford University Press

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A SMN missense mutation complements SMN2 restoring snRNPs and rescuing SMA mice

By Workman Eileen   Saieva Luciano   Carrel Tessa L.   Crawford Thomas O.   Liu Don   Lutz Cathleen   Beattie Christine E.   Pellizzoni Livio   Burghes Arthur H.M.  

Human Molecular Genetics, Vol. 18, Iss. 12, 2009-06 ,pp. : 2215-2229

Oxford University Press

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Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

By Dominguez Elisa   Marais Thibaut   Chatauret Nicolas   Benkhelifa-Ziyyat Sofia   Duque Sandra   Ravassard Philippe   Carcenac Romain   Astord Stphanie   de Moura Aurlie Pereira   Voit Thomas   Barkats Martine  

Human Molecular Genetics, Vol. 20, Iss. 4, 2011-02 ,pp. : 681-693

Oxford University Press

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Increasing expression and decreasing degradation of SMN ameliorate the spinal muscular atrophy phenotype in mice

By Kwon Deborah Y.   Motley William W.   Fischbeck Kenneth H.   Burnett Barrington G.  

Human Molecular Genetics, Vol. 20, Iss. 18, 2011-09 ,pp. : 3667-3677

Oxford University Press

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An exonic enhancer is required for inclusion of an essential exon in the SMA-determining gene SMN

By Lorson Christian L.   Androphy Elliot J.  

Human Molecular Genetics, Vol. 9, Iss. 2, 2000-01 ,pp. : 259-265

Oxford University Press

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