Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by mutations in the survival motor neuron 1 (<i>SMN2</i>) gene. All patients have at least one copy of a paralog, <i>SMN2</i>, but a C-to-T transition in this gene results in exon 7 skipping in a majority of tra...
| Hauptverfasser: | , , , , , , , , , , , |
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| Format: | Journal article |
| Sprache: | English |
| Veröffentlicht: |
Cell Press
2021
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