Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

Increasing survival of motor neuron 2, centromeric (SMN2) exon 7 inclusion to express more full-length SMN protein in motor neurons is a promising approach to treat spinal muscular atrophy (SMA), a genetic neurodegenerative disease. Previously, we identified a potent 2′-O-(2-methoxyethyl) (MOE) phos...

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Autori principali: Hua, Yimin, Sahashi, Kentaro, Hung, Gene, Rigo, Frank, Passini, Marco A., Bennett, C. Frank, Krainer, Adrian R.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Cold Spring Harbor Laboratory Press 2010
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Research Paper
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2912561/
https://ncbi.nlm.nih.gov/pubmed/20624852
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/gad.1941310
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