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Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the Exon
Several strategies have been pursued to increase the extent of exon 7 inclusion during splicing of SMN2 (survival of motor neuron 2) transcripts, for eventual therapeutic use in spinal muscular atrophy (SMA), a genetic neuromuscular disease. Antisense oligonucleotides (ASOs) that target an exon or i...
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| Main Authors: | , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Public Library of Science
2007
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC1820610/ https://ncbi.nlm.nih.gov/pubmed/17355180 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pbio.0050073 |
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