Molecular regulation of neural crest contribution in the developing cardiac outflow tract

Doctor's Name: 
G. Paul Matherne, MD
University of Virginia Health Sciences Center

This is an animal (mouse) gene therapy study in isolated heart tissue. Adenosine (a natural compound and a medicine) acting at A1 receptors has been shown to reduce myocardial injury due to ischemia (low blood flow) and hypoxia (low oxygen levels). A cardiac specific A1 receptor transgene has been introduced into the mouse genome producing a 1000 fold increase in functional A1 receptors in the heart. This study will attempt to determine whether this provides increased protection during the stressors noted above.

The significance of this project is that it supports a strategy for gene therapy in the heart. While this may be 5 - 10 years away from human clinical usage, this is how the process starts. This type of gene therapy will provide cardiologists and surgeons with another way to improve heart function after cardiopulmonary bypass, medical therapy, and surgery with patients with congenital heart disease with repeated exposure to ischemia and hypoxia.

As a result of CHF funding, Dr. Matherne was recently able to secure a million-dollar National Institute of Health research grant.

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