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...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Public Library of Science (PLoS)
2007-04-01
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| Series: | PLoS Biology |
| Online Access: | http://europepmc.org/articles/PMC1820610?pdf=render |