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Differences in wild-type– and R338L-tenase complex formation are at the root of R338L-factor IX assay discrepancies

Adeno-associated virus (AAV) gene therapy has the potential to functionally cure hemophilia B by restoring factor (F)IX concentrations into the normal range. Next-generation AAV therapies express a naturally occurring gain-of-function FIX variant, FIX-Padua (R338L-FIX), that increases FIX activity (...

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Detalles Bibliográficos
Autores principales:	Foley, Jonathan H., Shehu, Erald, Riddell, Anne, Gray, Elaine, Goodale, Andrew, Yu, I-Mei, Verhoef, Daniël, Little, John, Shattock, Diane, Kitchen, Steve, Chowdary, Pratima, Corbau, Romuald, Nathwani, Amit C.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	The American Society of Hematology 2022
Materias:	Thrombosis and Hemostasis
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9979717/ https://www.ncbi.nlm.nih.gov/pubmed/35839077 http://dx.doi.org/10.1182/bloodadvances.2022007435

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