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Muscle-specific SMN reduction reveals motor neuron–independent disease in spinal muscular atrophy models
Paucity of the survival motor neuron (SMN) protein triggers the oft-fatal infantile-onset motor neuron disorder, spinal muscular atrophy (SMA). Augmenting the protein is one means of treating SMA and recently led to FDA approval of an intrathecally delivered SMN-enhancing oligonucleotide currently i...
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| Pubblicato in: | J Clin Invest |
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| Autori principali: | , , , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
American Society for Clinical Investigation
2020
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7269591/ https://ncbi.nlm.nih.gov/pubmed/32039917 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI131989 |
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