Human Gene Therapy In the Department of Pediatrics

Key Documents

Mark A. Kay, M.D., Ph.D.

Academic Appointments

Contact Information

  • Academic Offices
    Personal Information
    Email Tel (650) 498-6531
    Administrative Contact
    Lorna Groundwater Administrative Associate Tel Work 650-498-6532

Professional Snapshot

Administrative Appointments

  • President, American Society of Gene Therapy (2005 - 2006)
  • President Elect, American Society of Gene Therapy (2004 - 2005)
  • Chair of Organizing Committee, Gordon Conference on Viral Vectors for Gene Therapy (2003 - 2004)
  • Vice President, ASGT (2003 - 2004)
  • Chief Scientific Advisor, Benitec, LLC (2003 - present)
  • View All 7administrative appointments of Mark Kay

Honors and Awards

  • Researcher of the Year, National Hemophilia Foundation (2000)
  • Pediatric Researcher of the Year, E. Mead Johnson Award (2000)
  • Elected Member, American Society for Clinical Investigation (1997)
  • Arosenius Swedish Honorary Lectureship, - (1997)
  • Young Investigator Award, Western Society for Clinical Investigation (1996)

Professional Education

B.S.: Michigan State University, Physical Sciences (1980)
Ph.D.: Case Western Reserve University, Developmental Genetics (1986)
M.D.: Case Western Reserve University, (1987)

Graduate & Fellowship Program Affiliations

Industry Relationships

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Consulting: Howrey Law Firm , Intradigm

Scientific Focus

Current Research Interests

The goal of the Program in Human Gene Therapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseases. The general approach is to develop new vector systems and delivery methods, test them in the appropriate animal models, uncover the mechanisms involved in vector transduction, and use the most promising approaches in clinical trials. Specifically, we work on a variety of viral and non-viral vector systems. Our major disease models are hemophilia, hepatitis C and B viral infections, and diabetes. The second major focus includes the role that small RNAs play in mammalian gene regulation.

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