Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts

By Mattis Virginia   Rai Ravi   Wang Jinhua   Chang Cheng-Wei   Coady Tristan   Lorson Christian  

Human Genetics, Vol. 120, Iss. 4, 2006-11 ,pp. : 589-601

Springer Publishing Company

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Early heart failure in the SMN7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

By Bevan Adam K.   Hutchinson Kirk R.   Foust Kevin D.   Braun Lyndsey   McGovern Vicki L.   Schmelzer Leah   Ward Jennifer G.   Petruska Jeffrey C.   Lucchesi Pamela A.   Burghes Arthur H.M.   Kaspar Brian K.  

Human Molecular Genetics, Vol. 19, Iss. 20, 2010-10 ,pp. : 3895-3905

Oxford University Press

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Reduced survival motor neuron ( Smn ) gene dose in mice leads to motor neuron degeneration: an animal model for spinal muscular atrophy type III

By Jablonka Sibylle   Schrank Bertold   Kralewski Martina   Rossoll Wilfried   Sendtner Michael  

Human Molecular Genetics, Vol. 9, Iss. 3, 2000-02 ,pp. : 341-346

Oxford University Press

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The role of the SMN gene in proximal spinal muscular atrophy

By Melki J.  

Human Molecular Genetics, Vol. 7, Iss. 10, 1998-10 ,pp. : 1531-1536

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Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model

By Mattis Virginia B.   Ebert Allison D.   Fosso Marina Y.   Chang Cheng-Wei   Lorson Christian L.  

Human Molecular Genetics, Vol. 18, Iss. 20, 2009-10 ,pp. : 3906-3913

Oxford University Press

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