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Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models
Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by the loss of Survival Motor Neuron-1 (SMN1). In all SMA patients, a nearly identical copy gene called SMN2 is present, which produces low levels of functional protein owing to an alternative splicing event. To prevent exon-skippin...
Αποθηκεύτηκε σε:
| Κύριοι συγγραφείς: | , , , , , , |
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| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
Oxford University Press
2014
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4140465/ https://ncbi.nlm.nih.gov/pubmed/24781211 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddu198 |
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