Exon skipping for SMN2: Toward a Treatment for SMA

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Jon Moulton
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Exon skipping for SMN2: Toward a Treatment for SMA

Mitrpant C, Porensky P, Zhou H, Price L, Muntoni F, Fletcher S, Wilton SD, Burghes AHM. Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy. PLoS ONE. 2013;8(4):e62114. doi:10.1371/journal.pone.0062114