Mark A. Kay, M.D., Ph.D.

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)

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)

Professional Education

Postdoctoral Advisees

Adi Barzel,  Hang Hang,  Lan Jin,  Leszek Lisowski,  Jiamiao Lu,  Qingjun Luo,  Biswajoy Roy-Chaudhuri,  Paul Valdmanis,  Shengchun Wang

Graduate & Fellowship Program Affiliations

• Genetics
• Pediatrics

Internet Links

• Gene Therapy

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

• The Extragenic Spacer Length Between the 5'' and 3'' Ends of the Transgene Expression Cassette Affects Transgene Silencing From Plasmid-based Vectors. Lu J, Zhang F, Xu S, Fire AZ, Kay MA. Mol Ther. 2012
• Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM. N Engl J Med. 2011; 365 (25): 2357-65
• Double knockdown of prolyl hydroxylase and factor-inhibiting hypoxia-inducible factor with nonviral minicircle gene therapy enhances stem cell mobilization and angiogenesis after myocardial infarction. Huang M, Nguyen P, Jia F, Hu S, Gong Y, de Almeida PE, Wang L, Nag D, Kay MA, Giaccia AJ, Robbins RC, Wu JC. Circulation. 2011; 124 (11 Suppl): S46-54
• Expression determinants of mammalian argonaute proteins in mediating gene silencing. Valdmanis PN, Gu S, Schuermann N, Sethupathy P, Grimm D, Kay MA. Nucleic Acids Res. 2011
• Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors. Narsinh KH, Jia F, Robbins RC, Kay MA, Longaker MT, Wu JC. Nat Protoc. 2011; 6 (1): 78-88