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Analysis of a read-through promoting compound in a severe mouse model of Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is the leading genetic cause of infantile death and caused by the loss of functional Survival Motor Neuron 1(SMN1). The remaining copy gene, SMN2, is unable to rescue from disease because the primary gene product lacks the final coding exon, exon 7, due to an alternativ...
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| Main Authors: | , , |
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| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
2012
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3426503/ https://ncbi.nlm.nih.gov/pubmed/22819971 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neulet.2012.07.024 |
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