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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...

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Detalles Bibliográficos
Publicado en:J Blood Med
Autores principales: Machin, Nicoletta, Ragni, Margaret V
Formato: Artigo
Lenguaje:Inglês
Publicado: Dove Medical Press 2018
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6110283/
https://ncbi.nlm.nih.gov/pubmed/30174468
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/JBM.S159297
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