Mark A. Kay, M.D., Ph.D.
Academic Appointments
- Professor, Pediatrics - Human Gene Therapy
- Member, Bio-X
- Member, Child Health Research Institute
- Member, Stanford Cancer Institute
- Professor, Genetics
Key Documents
Contact Information
- Academic Offices
Personal Information Email Tel (650) 498-6531Alternate Contact Melinda Hing Administrative Associate Email Tel Work 650-498-6532
Professional Overview
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 - 2005)
- Executive Committee, Faculty Senate - Stanford (2002 - present)
Honors and Awards
- Member, AAP (2010)
- 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) |
Postdoctoral Advisees
Adi Barzel, Qingjun Luo, Nicole Paulk, Biswajoy Roy-Chaudhuri, Lukasz Sobkowiak, Paul Valdmanis, Shengchun Wang, Yue Zhang
Graduate & Fellowship Program Affiliations
Internet Links
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.
Publications
- A Mini-intronic Plasmid (MIP): A Novel Robust Transgene Expression Vector In Vivo and In Vitro. Mol Ther. 2013; (5): 954-63
- Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Mol Ther. 2013; (1): 131-8
- The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA. Nucleic Acids Res. 2013; (6): 3688-98
- Expression determinants of mammalian argonaute proteins in mediating gene silencing. Nucleic Acids Res. 2012; (8): 3704-13
- Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging. Circ Res. 2012; (12): 1494-503
- Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. Mol Ther. 2012; (10): 1912-23
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