Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

Yimin Hua(Cold Spring Harbor Laboratory), Kentaro Sahashi(Cold Spring Harbor Laboratory), Frank Rigo(Ionis Pharmaceuticals (United States)), Gene Hung(Ionis Pharmaceuticals (United States)), Guy Horev(Cold Spring Harbor Laboratory), C. Frank Bennett(Ionis Pharmaceuticals (United States)), Adrian R. Krainer(Cold Spring Harbor Laboratory)
Nature
October 1, 2011
10.1038/nature10485
Cited by 740Open Access
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