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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
RNA interference via the endogenous miRNA pathway regulates gene expression by controlling protein synthesis through post-transcriptional gene silencing. In recent years, miRNA-mediated gene regulation has shown potential for treatment of neurological disorders caused by a toxic gain of function mec...
Autores principales: | Pourshafie, Naemeh, Lee, Philip R., Chen, Ke-lian, Harmison, George G., Bott, Laura C., Fischbeck, Kenneth H., Rinaldi, Carlo |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MyJove Corporation
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6126683/ https://www.ncbi.nlm.nih.gov/pubmed/30148479 http://dx.doi.org/10.3791/55724 |
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