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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...
保存先:
| 出版年: | Yonsei Med J |
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| 第一著者: | |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Yonsei University College of Medicine
2020
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7105407/ https://ncbi.nlm.nih.gov/pubmed/32233169 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3349/ymj.2020.61.4.273 |
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