160. Overcoming the Size Limitations of Gene Transfer Vectors: Using Segmental Trans-Splicing To Deliver the Coding Sequence of Von Willebrand Factor

Von Willebrand Disease (VWD), a common inherited bleeding disorder affecting 1 to 3% of the U.S. population, is caused by abnormalities in the expression and processing of von Willebrand factor (VWF), an adhesion molecule secreted by endothelial cells and platelets. Due to packaging limitations of c...

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Veröffentlicht in:	Molecular therapy 2004-05, Vol.9 (S1), p.S61-S61
Hauptverfasser:	Pergolizzi, Robert G, Dragos, Rachel, Nakayama, Katsutoshi, Wagner, Denisa, Crystal, Ronald G
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Biopsy Disease Dogs Drug dosages Gene therapy Genomes Hemophilia Medicine Mutagenesis Plasma Proteins Transplants & implants Vectors (Biology)
Online-Zugang:	Volltext
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