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RNA in spinal muscular atrophy: therapeutic implications of targeting
INTRODUCTION: Spinal muscular atrophy (SMA) is caused by low levels of the Survival Motor Neuron (SMN) protein due to deletions of or mutations in the SMN1 gene. Humans carry another nearly identical gene, SMN2, which mostly produces a truncated and less stable protein SMNΔ7 due to predominant skipp...
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| Publicado no: | Expert Opin Ther Targets |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7529864/ https://ncbi.nlm.nih.gov/pubmed/32538213 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/14728222.2020.1783241 |
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