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An investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B
Fitusiran is an RNA interference therapeutic that targets antithrombin (AT) in the liver and interferes with AT translation by binding and degrading messenger RNA-AT, thereby silencing AT gene expression and preventing AT synthesis. In both preclinical and clinical studies, AT knockdown results in d...
Autores principales: | Machin, Nicoletta, Ragni, Margaret V |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Dove Medical Press
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6110283/ https://www.ncbi.nlm.nih.gov/pubmed/30174468 http://dx.doi.org/10.2147/JBM.S159297 |
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