Correction of von Willebrand factor multimerization in type 2A/ⅡC von Willebrand disease by exogenous VWF propeptide supplementation
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Gene therapy remains the only cure for von Willebrand disease (VWD), but is limited by the large von Willebrand factor (VWF) gene size. Variants affecting the VWF propeptide (VWFpp) impair multimerization, causing type 2A/IIC VWD. VWFpp serves as a pH-sensitiv... ...
