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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Bibliographic Details
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
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2012-01-01
Series:PLoS ONE
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/22973456/?tool=EBI