Glembotski Lab   >>   Research   >>   Gene Therapy

Regenerative Medicine and Stem Cells:

One of the main goals of research in the Glembotski lab is to develop prototypes of therapies that could be used as platforms for the development of novel treatments for heart disease. One such prototype involves the selective delivery of cardioprotective genes to the heart using an adenovirus-associated (AAV) gene therapy approach. In our studies of secretion, ER stress and stem cells in the heart, we have completed microarray analyses to identify mRNAs and microRNAs that are regulated in response to protective stress signaling pathways. As a result of those arrays, we have identified candidate genes that have been selected for development of prototypes for gene therapy. We have packaged the candidate genes into AAV9, a form of AAV that preferentially delivers genes to the heart. Moreover, we have engineered these genes so that their expression is regulated by cardiac-specific promoters, which further enhancing their utility and therapeutic safety. We have already shown the cardioprotective effects of several candidate genes using the AAV9 gene therapy approach, thus demonstrating the therapeutic potential of this platform for treating heart disease.

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