Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor.

@article{Meyer2006PhenotypicCO,
  title={Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor.},
  author={Simon F De Meyer and Karen Vanhoorelbeke and Marinee K. L. Chuah and Inge M Pareyn and Veerle Gillijns and Robert P. Hebbel and D{\'e}sir{\'e} Collen and Hans Deckmyn and Thiery C Vandendriessche},
  journal={Blood},
  year={2006},
  volume={107 12},
  pages={
          4728-36
        }
}
Von Willebrand disease (VWD) is an inherited bleeding disorder, caused by quantitative (type 1 and 3) or qualitative (type 2) defects in von Willebrand factor (VWF). Gene therapy is an appealing strategy for treatment of VWD because it is caused by a single gene defect and because VWF is secreted into the circulation, obviating the need for targeting specific organs or tissues. However, development of gene therapy for VWD has been hampered by the considerable length of the VWF cDNA (8.4 kb… CONTINUE READING

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Management of VWD.

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Immune responses to AAV and to factor IX in a phase I study of AAV - mediated , liver - directed gene transfer for hemophilia B

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