Development of Methods for the Selective Measurement of the Single Amino Acid Exchange Variant Coagulation Factor IX Padua

The description of hyper-functional factor IX (FIX) Padua triggered the development of BAX 335, an AAV8-based hemophilia B gene therapy vector designed to compensate for low FIX protein expression levels by expressing the FIX Padua variant, thereby reducing the exposure to viral vector. The presence...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Weber, Alfred, Engelmaier, Andrea, Voelkel, Dirk, Pachlinger, Robert, Scheiflinger, Friedrich, Monahan, Paul E., Rottensteiner, Hanspeter
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6039963/
https://ncbi.nlm.nih.gov/pubmed/30003118
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.05.004
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