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Blocking p62-dependent SMN degradation ameliorates spinal muscular atrophy disease phenotypes
Spinal muscular atrophy (SMA), a degenerative motor neuron (MN) disease, caused by loss of functional survival of motor neuron (SMN) protein due to SMN1 gene mutations, is a leading cause of infant mortality. Increasing SMN levels ameliorates the disease phenotype and is unanimously accepted as a th...
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| Veröffentlicht in: | J Clin Invest |
|---|---|
| Hauptverfasser: | , , , , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
American Society for Clinical Investigation
2018
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6025996/ https://ncbi.nlm.nih.gov/pubmed/29672276 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI95231 |
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