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Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA
BACKGROUND: Spinal Muscular Atrophy (SMA) is the leading genetic cause of infantile death. It is caused by the loss of functional Survival Motor Neuron 1 (SMN1). There is a nearly identical copy gene, SMN2, but it is unable to rescue from disease due to an alternative splicing event that excises a n...
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| Main Authors: | , , , |
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| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
BioMed Central
2009
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2789732/ https://ncbi.nlm.nih.gov/pubmed/19948047 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1471-2202-10-142 |
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