Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)
Von Willebrand disease is an inherited bleeding disorder that impacts the blood's ability to clot properly. Von Willebrand disease is cause by the lack or not working substance in the blood known as Von Willebrand factor.
Current therapy for Von Willebrand disease includes desmopressin acetate (DDAVP) and /or VWF/FVIII concentrates. Patients with severe Von Willebrand disease face a lifetime of weekly treatments and mounting medical bills. Gene therapy could help these patients improve their quality of life by providing the missing factors necessary for the blood's ability to clot properly.
The gene transfer options being studied include naked DNA, viral gene transfer vectors encoding Von Willebrand factor transgenes, and ex vivo cell therapy. The latter involves transplantation of the patient's own cells modified with a corrected copy of the defective gene. Human blood outgrowth endothelial cells (BOEC) display all the properties needed for successful ex vivo cell therapy. We plan to obtain blood samples from normal research subjects and patients with Von Willebrand Disease in order to isolate blood outgrowth endothelial cells (BOEC) from peripheral blood, and develop a ex vivo gene therapy for Von Willebrand Disease.
Von Willebrand Disease
|Study Design:||Observational Model: Case Control
Time Perspective: Cross-Sectional
|Official Title:||Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)|
|Study Start Date:||November 2008|
|Estimated Study Completion Date:||October 2010|
|Estimated Primary Completion Date:||July 2010 (Final data collection date for primary outcome measure)|
Control Group. Normal (healthy) individuals without Von Willebrand Disease.
Case Group. Individuals with known Von Willebrand Disease.
|United States, New York|
|Weill Cornell Medical College|
|New York, New York, United States, 10021|
|Principal Investigator:||Ronald G Crystal||Weill Medical College of Cornell University|