Splice-switching therapy for spinal muscular atrophy.
Spinal muscular atrophy (SMA) is a genetic disorder with severity ranging from premature death in infants to restricted motor function in adult life. Despite the genetic cause of this disease being known for over twenty years, only recently has a therapy been approved to treat the most severe form o...
| Hauptverfasser: | , , |
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| Format: | Journal article |
| Sprache: | English |
| Veröffentlicht: |
MDPI
2017
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