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Recent Advances in Antisense Oligonucleotide Therapy in Genetic Neuromuscular Diseases
Genetic neuromuscular diseases are caused by defective expression of nuclear or mitochondrial genes. Mutant genes may reduce expression of wild-type proteins, and strategies to activate expression of the wild-type proteins might provide therapeutic benefits. Also, a toxic mutant protein may cause ce...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Medknow Publications & Media Pvt Ltd
2018
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5909143/ https://www.ncbi.nlm.nih.gov/pubmed/29720791 http://dx.doi.org/10.4103/aian.AIAN_298_17 |