My laboratory is presently involved in three major areas of research. The first involves gene therapy targeted primarily to liver. In particular, we focus on non-viral approaches including gene repair via single-stranded oligonucleotides, and the Sleeping Beauty transposon system. Our clinical…
more targets include hemophilia, Crigler-Najjar Syndrome, ornithine transcarbamylase deficiency, sickle cell anemia and others. In our second area of research, we have discovered that ursodeoxycholic acid, an endogenous hydrophilic bile acid, is a potent antiapoptotic agent. We have characterized the effects and mechanism by which ursodeoxycholic acid, as well as its taurine conjugate, functions to inhibit apoptosis. We have studied several animal models that are relatively accurate to their human counterparts, including transgenic models of Huntington’s disease, acute ischemic and hemorrhagic stroke and cell transplantation for Parkinson’s disease. Our third area of research involves the function, biogenesis and profiling of microRNAs in several different disease entities, including colon and hepatocellular cancer. In addition, we have active programs characterizing microRNAs in liver regeneration, stem cells and mitochondrial function.
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