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Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive disease affecting ∼1 in 10,000 live births. The most striking component is the loss of α-motor neurons in the ventral horn of the spinal cord, resulting in progressive paralysis and eventually premature death. There is no current treatment para...
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| Main Authors: | , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Mary Ann Liebert, Inc.
2013
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3655628/ https://ncbi.nlm.nih.gov/pubmed/23544870 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.225 |
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