Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants

The high-specific-activity factor IX (FIX) variant Padua (R338L) is the most promising transgene for hemophilia B (HB) gene therapy. Although R338 is strongly conserved in mammalian evolution, amino acid substitutions at this position are underrepresented in HB databases. We therefore undertook a co...

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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Samelson-Jones, Benjamin J., Finn, Jonathan D., Raffini, Leslie J., Merricks, Elizabeth P., Camire, Rodney M., Nichols, Timothy C., Arruda, Valder R.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2021
Assuntos:
Thrombosis and Hemostasis
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7948292/
https://ncbi.nlm.nih.gov/pubmed/33656538
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2019000405
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