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Spinal muscular atrophy: from tissue specificity to therapeutic strategies
Spinal muscular atrophy (SMA) is the most frequent genetic cause of death in infants and toddlers. All cases of spinal muscular atrophy result from reductions in levels of the survival motor neuron (SMN) protein, and so SMN upregulation is a focus of many preclinical and clinical studies. We examine...
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| Publicado no: | F1000Prime Rep |
|---|---|
| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Faculty of 1000 Ltd
2015
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4311279/ https://ncbi.nlm.nih.gov/pubmed/25705387 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.12703/P7-04 |
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