Gene and Cell Based Therapies

We are developing gene therapy approaches that target the contractile motor protein myosin and the troponin protein complex that controls turning contraction on and off.  These gene therapies are tested on muscle cells in culture and in animal models of heart or skeletal muscle disease or myocardial infarct (heart attack).  Cell based therapies involve engineering human inducible pluripotent stem cell derived cardiomyocytes (hiPSC-CM) that have increased contractile performance and can also deliver a nucleotide that increases contraction to the host myocardium when these hiPSC-CMs are transplanted to a damaged or weakened heart.

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