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Antisense Oligonucleotide-Mediated Exon-skipping Therapies: Precision Medicine Spreading from Duchenne Muscular Dystrophy
In 1995, we were the first to propose antisense oligonucleotide (ASO)-mediated exon-skipping therapy for the treatment of Duchenne muscular dystrophy (DMD), a noncurable, progressive muscle-wasting disease. DMD is caused by deletion mutations in one or more exons of the DMD gene that shift the trans...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Japan Medical Association
2021
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8355726/ https://www.ncbi.nlm.nih.gov/pubmed/34414317 http://dx.doi.org/10.31662/jmaj.2021-0019 |