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Mechanistic studies of a small-molecule modulator of SMN2 splicing
RG-7916 is a first-in-class drug candidate for the treatment of spinal muscular atrophy (SMA) that functions by modulating pre-mRNA splicing of the SMN2 gene, resulting in a 2.5-fold increase in survival of motor neuron (SMN) protein level, a key protein lacking in SMA patients. RG-7916 is currently...
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| Publicado no: | Proc Natl Acad Sci U S A |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
National Academy of Sciences
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5960314/ https://ncbi.nlm.nih.gov/pubmed/29712837 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1800260115 |
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