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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...
| Autores principales: | , , , |
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| Formato: | Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2009
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2789732/ https://www.ncbi.nlm.nih.gov/pubmed/19948047 http://dx.doi.org/10.1186/1471-2202-10-142 |