Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)

22:16 EDT 28th May 2015 | BioPortfolio

Summary

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.

Study Design

Observational Model: Case Control, Time Perspective: Cross-Sectional

Conditions

Von Willebrand Disease

Location

Weill Cornell Medical College
New York
New York
United States
10021

Status

Active, not recruiting

Source

Weill Medical College of Cornell University

Results (where available)

View Results

Links

Clinical Trials [22 Associated Clinical Trials listed on BioPortfolio]

Phase I Study of Human Von Willebrand Factor for Von Willebrand's Disease

OBJECTIVES: I. Evaluate the effect of a new von Willebrand factor concentrate on bleeding time, in vivo recovery, and circulating half-life of the infused factor in patients with von Will...

National Study of Moderate and Severe Von Willebrand Disease in the Netherlands

The objective of this study is to assess the clinical presentation, the treatment and the complications of the disease and treatment in moderate and severe von Willebrand disease. Another ...

Phase II Study of IL-11 (Neumega) in Von Willebrand Disease

This study is testing the use of rhIL-11 (recombinant interleukin 11, Neumega) in individuals with Von Willebrand disease. The purpose is to evaluate: 1. if rhIL-11 corrects VWF (Von Wi...

Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease

The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of ...

The Von Willebrand Disease (VWD) International Prophylaxis Study

The von Willebrand Disease Prophylaxis Network (vWD PN) is an international study group formed with the goal of investigating the role of prophylaxis in clinically severe VWD that is non-r...

PubMed Articles [14737 Associated PubMed Articles listed on BioPortfolio]

Determination of von Willebrand factor multimers in Mexican population.

Background: Von Willebrand disease is an inherited disease in which the structure, function, and concentration of von Willebrand factor are altered, as well as the platelet von Willebrand factor endot...

Von Willebrand Factor Deficiency Corrected by Lung Transplantation.

In experimental models with von Willebrand disease pigs, plasma von Willebrand factor (vWF) was significantly increased after lung transplantation because lung endothelial cells strongly express vWF. ...

Clinical use of Haemate(®) P in von Willebrand disease: A 25-year retrospective observational study.

von Willebrand disease (VWD) is caused by dysfunction or diminished levels of von Willebrand factor (VWF). VWF-containing plasma concentrates are used for treatment of patients with VWD for whom desmo...

Interactions of von Willebrand factor and ADAMTS13 in von Willebrand disease and thrombotic thrombocytopenic purpura.

The function of von Willebrand factor (VWF), a huge multimeric protein and a key factor in platelet dependent primary haemostasis, is regulated by its specific protease ADAMTS13. The ADAMTS13 dependen...

Changes in von Willebrand factor level and von Willebrand activity with age in type 1 von Willebrand disease.

In a normal population, VWF plasma levels (VWF:Ag) and VWF activity (VWF:RCo) increase by approximately 0.17 and 0.15 IU mL(-1) per decade, but the influence of age is unknown in patients with type ...

Medical and Biotech [MESH] Definitions

A subtype of von Willebrand disease that results from a partial deficiency of VON WILLEBRAND FACTOR.

A subtype of von Willebrand disease that results from qualitative deficiencies of VON WILLEBRAND FACTOR. The subtype is divided into several variants with each variant having a distinctive pattern of PLATELET-interaction.

A subtype of von Willebrand disease that results from a total or near total deficiency of VON WILLEBRAND FACTOR.

A high-molecular-weight plasma protein, produced by endothelial cells and megakaryocytes, that is part of the factor VIII/von Willebrand factor complex. The von Willebrand factor has receptors for collagen, platelets, and ristocetin activity as well as the immunologically distinct antigenic determinants. It functions in adhesion of platelets to collagen and hemostatic plug formation. The prolonged bleeding time in VON WILLEBRAND DISEASES is due to the deficiency of this factor.

Group of hemorrhagic disorders in which the VON WILLEBRAND FACTOR is either quantitatively or qualitatively abnormal. They are usually inherited as an autosomal dominant trait though rare kindreds are autosomal recessive. Symptoms vary depending on severity and disease type but may include prolonged bleeding time, deficiency of factor VIII, and impaired platelet adhesion.

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