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

2014-08-27 03:32:24 | 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: 2014-08-27T03:32:24-0400

Clinical Trials [38 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 ...

rVWF IN PROPHYLAXIS

The purpose of this phase 3 study is to investigate the efficacy and safety, including immunogenicity and thrombogenicity of prophylactic treatment with recombinant von Willebrand factor (...

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 ...

PubMed Articles [14206 Associated PubMed Articles listed on BioPortfolio]

Feasibility of the Von Willebrand disease PREVENT trial.

Despite treatment, women with von Willebrand disease (VWD) have lower von Willebrand factor (VWF) levels and greater blood loss at delivery than controls. Current weight-based dosing does not account ...

Clinical and Laboratory Phenotype Variability in Type 2M Von Willebrand Disease.

Von Willebrand factor (VWF) is a multimeric coagulation factor that tethers platelets to injured subendothelium. Type 2M von Willebrand Disease (VWD) is characterized by a qualitative defect in VWF wi...

Von Willebrand disease type 3 falsely diagnosed as hemophilia A: a case report.

The von Willebrand disease type 3 can be confused with hemophilia A because a very low level of factor VIIIC, we will report the case of patient with von Willebrand disease type 3 treated as moderate ...

Abnormal von Willebrand factor secretion, factor VIII stabilization, and thrombus dynamics in type 2N von Willebrand disease mice.

von Willebrand factor (VWF) and factor VIII (FVIII) circulate as a non-covalent complex with VWF serving as the carrier for FVIII. VWF indirectly influences secondary hemostasis by stabilizing FVIII a...

Revascularization operation for moyamoya disease with concurrent von Willebrand disease: A case report.

Although extracranial-intracranial (EC-IC) bypass is an effective treatment strategy for symptomatic moyamoya disease, surgeons need to be cautious regarding the possibility of postoperative hemorrhag...

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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