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...
| Main Authors: | , , , , , , , , , , , |
|---|---|
| 格式: | Journal article |
| 语言: | English |
| 出版: |
Cell Press
2021
|