Molecular Genetics and Gene Therapy of Cardiovascular Disease
The
laboratory is actively involved in developing new genetic therapies
for cardiovascular disease. In the area of cardiomyopathy, we are studying
gene replacement in an autosomal recessive form of fatal cardiomyopathy
in children. The disease is the prototype of lysosomal storage disorders
leading to skeletal and cardiac muscle weakness. We have used AAV vectors
to achieve sustained correction of the gene deficiency and correction
of the phenotype in natural and transgenic mouse models of the disease.
The current therapy is currently being proposed for human clinical trials.
Similar therapies are being used to combat cardiac transplantation rejection.
Secondly, we are investigating the ability of mescnchymal stem cells
to undergo myocardial specification for the purpose of tissue repair
in the heart. Finally, several projects are focused on the use of AAV
vectors injected into striated muscle to achieve sustained release of
therapeutic proteins, including thrombolytic factors and coagulation
factors.
These projects are currently funded by the NIH, AHA, and foundation
grants.
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