siRNA silencing of calumenin enhances functional factor IX production

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siRNA silencing of calumenin enhances functional factor IX production.

To improve production of functional fully gamma-carboxylated recombinant human clotting factor IX (r-hFIX), cell lines stably overexpressing r-hFIX have been engineered to also overexpress proteins of the gamma-carboxylation system. Here we demonstrate that siRNA silencing of calumenin, an inhibitor of the gamma-carboxylation system, enhances production of functional r-hFIX produced by engineer...

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Structural and functional basis of the developmental regulation of human coagulation factor IX gene: factor IX Leyden.

Hemophilia B Leyden is characterized by unusual developmental regulation of factor IX synthesis in affected individuals. One family affected with the hemophilia B Leyden phenotype was found to have a specific single-base mutation (G----A) at nucleotide -6 of the factor IX gene. The mutation site was found in a small region of the 5'-untranslated sequence designated the Leyden-specific region (L...

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ژورنال

عنوان ژورنال: Blood

سال: 2006

ISSN: 0006-4971,1528-0020

DOI: 10.1182/blood-2006-02-004671