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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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| Publicado no: | Ann Indian Acad Neurol |
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| Autor principal: | |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Medknow Publications & Media Pvt Ltd
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5909143/ https://ncbi.nlm.nih.gov/pubmed/29720791 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4103/aian.AIAN_298_17 |
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