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Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model
Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality and is caused by the loss of a functional SMN1 gene. In humans, there exists a nearly-identical copy gene known as SMN2 that encodes an identical protein as SMN1, but differs by a silent C to T transition within exon 7. T...
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| Main Authors: | , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
Oxford University Press
2009
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2748896/ https://ncbi.nlm.nih.gov/pubmed/19625298 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddp333 |
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