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Allele-Specific Gene Silencing in Two Mouse Models of Autosomal Dominant Skeletal Myopathy
We explored the potential of mutant allele-specific gene silencing (ASGS) in providing therapeutic benefit in two established mouse models of the autosomal dominantly-inherited muscle disorders, Malignant Hyperthermia (MH) and Central Core Disease (CCD). Candidate ASGS siRNAs were designed and valid...
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| Главные авторы: | , , , , |
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| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
Public Library of Science
2012
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3495761/ https://ncbi.nlm.nih.gov/pubmed/23152933 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0049757 |
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