Mark A. Kay

Last updated

Mark A. Kay
Born
Mark Allan Kay
NationalityAmerican
Alma mater Michigan State University (B.S.)
Case Western Reserve University (M.D., Ph.D.)
Known for AAV vector biology, liver-directed gene therapy, RNA interference
Awards National Academy of Inventors (2020)
ASGCT Outstanding Investigator Award (2013)
E. Mead Johnson Award (2000)
National Hemophilia Foundation Researcher of the Year Award (2000)
Scientific career
Fields Gene therapy, RNA biology, Genome editing
Institutions Stanford University

Mark A. Kay is an American physician-scientist recognized for his contributions to the field of gene therapy, genome editing, and RNA biology. He has an h-index of 123, and is known for liver-directed gene therapy, demonstrating RNA interference in mammals, and advancing adeno-associated virus (AAV) vector biology. [1] [2]

Contents

Education

Kay earned his B.S. in Physical Sciences from Lyman Briggs College at Michigan State University in 1980, and his M.D. and Ph.D. in Developmental Genetics from Case Western Reserve University in 1987. He completed a pediatric residency, medical genetics fellowship, and postdoctoral fellowship at Baylor College of Medicine in 1993. [3]

Career and research

Kay began his work in gene therapy in 1990. He was a founding member of the American Society of Gene and Cell Therapy (ASGCT) in 1996 and served as its Vice President, President-Elect, and President from 2003 to 2006. He played a role in establishing the society’s journal, Molecular Therapy , and advancing gene therapy research during a period of skepticism. [4]

Kay has been a long-time editor of Human Gene Therapy . His research has led to advances in gene transfer mechanisms, vector biology, and gene regulation. He demonstrated the correction of Hemophilia B in a large animal model and filed the initial Investigational New Drug (IND) application for systemic administration of recombinant AAV in humans. [5] [6]

Kay’s team demonstrated functional RNA interference (RNAi) in whole mammals. His research has also explored microRNA (miRNA)-mediated gene repression, tRNA-derived small RNAs, and their applications in cancer therapeutics. [7]

Academic and professional roles

From 1993 to 1998, Kay was a faculty member at the University of Washington in the Division of Medical Genetics, Department of Medicine. In 1998, he was recruited to Stanford University, where he currently serves as the Dennis Farrey Family Professor in the Departments of Pediatrics and Genetics and as Head of the Division of Human Gene Therapy in Pediatrics. [4]

He co-founded Avocel Inc., Voyager Therapeutics, and LogicBio Therapeutics. [8]

Honors and recognition

Kay was elected to the National Academy of Inventors in 2020. He received the ASGCT Outstanding Investigator Award (2013), the E. Mead Johnson Award for Pediatric Research (2000), and the National Hemophilia Foundation Researcher of the Year Award (2000). He has mentored over 60 scientists. [9]

Research and publications

Kay has published nearly 300 research articles, including over 30 in high-impact journals such as Nature , Science , Cell , and The New England Journal of Medicine . His work has been cited over 65,000 times, and he has an h-index of 123. [10]

Selected publications

References

  1. "Mark A. Kay, MD, PhD". Stanford Medicine . Retrieved April 17, 2025.
  2. Lisjak, Michela; De Caneva, Alessia; Marais, Thibaut; Barbon, Elena; Biferi, Maria Grazia; Porro, Fabiola; Barzel, Adi; Zentilin, Lorena; Kay, Mark A.; Mingozzi, Federico; Muro, Andrés F. (March 11, 2022). "Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice". Frontiers in Genome Editing. 4. doi: 10.3389/fgeed.2022.785698 . PMC   8962648 . PMID   35359664.
  3. "Kay Lab". Stanford Medicine . Retrieved April 17, 2025.
  4. 1 2 "Mark A. Kay, MD, PhD – Kriya Therapeutics". Kriya Therapeutics. April 8, 2024. Retrieved April 17, 2025.
  5. "Mark A. Kay, M.D., Ph.D. – Tenaya Therapeutics". Tenaya Therapeutics. Retrieved April 17, 2025.
  6. Kay, Mark A.; Rothenberg, Steven; Landen, Charles N.; Bellinger, Dwight A.; Leland, Frances; Toman, Carol; Finegold, Milton; Thompson, Arthur R.; Read, M. S.; Brinkhous, Kenneth M.; Woo, Savio L. C. (October 1993). "In Vivo Gene Therapy of Hemophilia B: Sustained Partial Correction in Factor IX-Deficient Dogs" . Science . 262 (5130): 117–119. Bibcode:1993Sci...262..117K. doi:10.1126/science.8211118. PMID   8211118.
  7. "Mark A. Kay profile – Springer Nature". Springer Nature . Retrieved April 17, 2025.
  8. "Gene Editing Technology". Drug Development & Delivery. March 2022. Retrieved April 17, 2025.
  9. "Gene Therapy Pioneer Dr. Mark A. Kay Joins Scientific Advisory Board". BusinessWire . Retrieved April 17, 2025.
  10. "Mark A. Kay – Google Scholar". Google Scholar. Retrieved April 17, 2025.