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

2014-08-26 22:32:24 | 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

Published on BioPortfolio: 2014-08-26T22:32:24-0400

Clinical Trials [23 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 [14962 Associated PubMed Articles listed on BioPortfolio]

Maxillary pseudotumor as initial manifestation of von Willebrand disease, type 2: report of a rare case and literature review.

Von Willebrand disease (VWD) is a bleeding disorder associated with inherited defects of von Willebrand factor (VWF). Type 2 N VWD is characterized by impaired FVIII-binding capacity (VWF:FVIIIB). Pse...

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. ...

Effect of the Novel Biodegradable N, O-Carboxymethylchitosan and Oligo-Chitosan on the Platelet Thrombogenicity Cascade in von Willebrand Disease.

Von Willebrand disease (vWD) is the second least common hemostatic disorder in Malaysia, and it has a low prevalence. This study examined the underlying platelet thrombogenicity cascades in the presen...

Clinical phenotype in genetically confirmed von Willebrand disease type 2N patients reflects a haemophilia A phenotype.

Von Willebrand disease (VWD) type 2N is characterized by a defective binding of factor VIII (FVIII) to von Willebrand factor (VWF) resulting in diminished plasma FVIII levels and a clinical phenotype ...

Angiogenic characteristics of blood outgrowth endothelial cells from patients with von Willebrand disease.

Endothelial von Willebrand factor (VWF) inhibits angiogenesis. Accordingly, blood outgrowth endothelial cells (BOECs) isolated from von Willebrand disease (VWD) patients showed enhanced in vitro angio...

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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