Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.
Hua, Yimin; Sahashi, Kentaro; Rigo, Frank; Hung, Gene; Horev, Guy; Bennett, C. Frank; Krainer, Adrian R.; Nature, 2011, vol. 478, issue 7367, p 123, ISSN 00280836. ISBN 00280836.

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