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Mark Kay, Ph.D.
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Mark Kay, Ph.D.

Mark A. Kay MD, PhD, Dennis Farrey Family Professor, Department of Pediatrics; Professor, Department of Genetics, Stanford University School of Medicine
       Research Description: Dr. Kay’s research for over 25 years is related to gene therapy and non-coding RNA biology and he is an acknowledged leader in this field. He has long been interested in the development of gene transfer vectors for gene therapy as well as manipulating non-coding RNAs for therapeutic purposes. His foundational studies have led to several multi-institutional human trials for gene therapy of conditions like hemophilia. A major interest has been in unraveling the mechanism of viral vector transduction in vivo. His work during the last 15 years has focused on two vector systems, mini-circles and recombinant AAVs (rAAV). Kay’s group has shown that DNA vectors consisting of a circularized eukaryotic expression cassette (lacking plasmid DNA backbone sequences) provide more persistent levels of transgene expression from quiescent tissues compared to the same plasmid vectors. Dr. Kay’s laboratory uncovered insights into how an Adenoviral vector expressing beta-cell transcription factors results in reprogramming of non-beta cells into functional beta- like cells in vivo. Dr. Kay has been an active member of the diabetes and islet biology community, with funded projects with Dr. Markus Grompe previously in the NIH/NIDDK Beta Cell Biology Consortium, and currently is a co-PI on a UO1 project of the Human Islet Resource Network in the Consortium for Targeting and Regeneration (CTAR). Their group is currently investigating mechanisms and methods to develop gene transfer vectors that when delivered by intravenous infusion target native islet cells, or re-program liver cells into functional human beta-cells.
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Mark A. Kay MD, PhD, Dennis Farrey Family Professor, Department of Pediatrics; Professor, Department of Genetics, Stanford University School of Medicine

Research Description: Dr. Kay’s research for over 25 years is related to gene therapy and non-coding RNA biology and he is an acknowledged leader in this field. He has long been interested in the development of gene transfer vectors for gene therapy as well as manipulating non-coding RNAs for therapeutic purposes. His foundational studies have led to several multi-institutional human trials for gene therapy of conditions like hemophilia. A major interest has been in unraveling the mechanism of viral vector transduction in vivo. His work during the last 15 years has focused on two vector systems, mini-circles and recombinant AAVs (rAAV). Kay’s group has shown that DNA vectors consisting of a circularized eukaryotic expression cassette (lacking plasmid DNA backbone sequences) provide more persistent levels of transgene expression from quiescent tissues compared to the same plasmid vectors. Dr. Kay’s laboratory uncovered insights into how an Adenoviral vector expressing beta-cell transcription factors results in reprogramming of non-beta cells into functional beta- like cells in vivo. Dr. Kay has been an active member of the diabetes and islet biology community, with funded projects with Dr. Markus Grompe previously in the NIH/NIDDK Beta Cell Biology Consortium, and currently is a co-PI on a UO1 project of the Human Islet Resource Network in the Consortium for Targeting and Regeneration (CTAR). Their group is currently investigating mechanisms and methods to develop gene transfer vectors that when delivered by intravenous infusion target native islet cells, or re-program liver cells into functional human beta-cells.

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