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|Porensky PN, Mitrpant C, McGovern VL, et al. (2012) A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse. Human Molecular Genetics. 21: 1625-38|
|Bevan AK, Hutchinson KR, Foust KD, et al. (2010) Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery. Human Molecular Genetics. 19: 3895-905|
|Foust KD, Wang X, McGovern VL, et al. (2010) Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nature Biotechnology. 28: 271-4|