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Mechanistic principles of antisense targets for the treatment of Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a major neurodegenerative disorder of children and infants. SMA is primarily caused by low levels of SMN protein owing to deletions or mutations of the survival motor neuron 1 (SMN1) gene. SMN2, a nearly identical copy of SMN1, fails to compensate for the loss of the...
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| Publicat a: | Future Med Chem |
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| Autors principals: | , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
2015
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4660980/ https://ncbi.nlm.nih.gov/pubmed/26381381 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4155/fmc.15.101 |
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