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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Detalles Bibliográficos
Autores principales: Weber, Alfred, Engelmaier, Andrea, Voelkel, Dirk, Pachlinger, Robert, Scheiflinger, Friedrich, Monahan, Paul E., Rottensteiner, Hanspeter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6039963/
https://www.ncbi.nlm.nih.gov/pubmed/30003118
http://dx.doi.org/10.1016/j.omtm.2018.05.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6039963/
https://www.ncbi.nlm.nih.gov/pubmed/30003118
http://dx.doi.org/10.1016/j.omtm.2018.05.004

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