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A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse
Spinal muscular atrophy (SMA) is an autosomal-recessive disorder characterized by α-motor neuron loss in the spinal cord anterior horn. SMA results from deletion or mutation of the Survival Motor Neuron 1 gene (SMN1) and retention of SMN2. A single nucleotide difference between SMN1 and SMN2 results...
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| Main Authors: | , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Oxford University Press
2012
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3298284/ https://ncbi.nlm.nih.gov/pubmed/22186025 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddr600 |
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