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Nusinersen as a Therapeutic Agent for Spinal Muscular Atrophy
The reduction of survival motor neuron (SMN) protein causes spinal muscular atrophy (SMA), an autosomal recessive neuromuscular disease. Nusinersen is an antisense oligonucleotide, approved by the FDA, which specifically binds to the repressor within SMN2 exon 7 to enhance exon 7 inclusion and augme...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Yonsei University College of Medicine
2020
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7105407/ https://www.ncbi.nlm.nih.gov/pubmed/32233169 http://dx.doi.org/10.3349/ymj.2020.61.4.273 |