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Trans-Splicing-Mediated Improvement in a Severe Mouse Model of Spinal Muscular Atrophy
Spinal muscular atrophy is a leading genetic cause of infantile death and occurs in ∼1/6000 live births. SMA is caused by the loss of Survival Motor Neuron-1 (SMN1), however, all patients retain at least one copy of a nearly identical gene called SMN2. While SMN2 and SMN1 are comprised of identical...
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| Main Authors: | , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Society for Neuroscience
2010
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| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2836862/ https://ncbi.nlm.nih.gov/pubmed/20053895 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1523/JNEUROSCI.4489-09.2010 |
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