Kentaro Sahashi, Karen K. Y. Ling, Yimin Hua, John Erby Wilkinson, Tomoki Nomakuchi, Frank Rigo, Gene Hung, David Xu, Ya-Ping Jiang, Richard Z. Lin, Chien-Ping Ko, C. Frank Bennett and Adrian R. Krainer Pathological impact of SMN2 mis-splicing in adult SMA mice EMBO Molecular Medicine 5
Article first published online: 9 SEP 2013 | DOI: 10.1002/emmm.201302567
An SMA mouse model mice mimicking adult-onset SMA was generated, witha characteristic late-onset motor phenotype. This study also suggests that high SMN levels are not required in adults, and that there is a broad therapeutic time window for treatment of adult-onset SMA.
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