Chemical chaperones improve protein secretion and rescue mutant factor VIII in mice with hemophilia A.
Inefficient intracellular protein trafficking is a critical issue in the pathogenesis of a variety of diseases and in recombinant protein production. Here we investigated the trafficking of factor VIII (FVIII), which is affected in the coagulation disorder hemophilia A. We hypothesized that chemical...
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Main Authors: | Stefanie D Roth, Jörg Schüttrumpf, Peter Milanov, Daniela Abriss, Christopher Ungerer, Patricia Quade-Lyssy, Jeremy C Simpson, Rainer Pepperkok, Erhard Seifried, Torsten Tonn |
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Format: | article |
Language: | EN |
Published: |
Public Library of Science (PLoS)
2012
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Online Access: | https://doaj.org/article/c3ff58452a9c4d699c82179d32d05aaf |
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