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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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| Hlavní autoři: | , , , , , , |
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| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
Cold Spring Harbor Laboratory Press
2010
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| Témata: | |
| On-line přístup: | 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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