Haiyan Fu, Ph.D. :: Nationwide Children's Hospital, Columbus, Ohio

Haiyan Fu, PhD

Haiyan  Fu, PhD

Center for Gene Therapy
Principal Investigator

Contact Information

The Research Institute at Nationwide Children's Hospital
700 Childrens Drive
Columbus, OH 43205 [ map ]
PH: (614) 722-5615
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Haiyan Fu, PhD, is a principal investigator in the Center for Gene Therapy at the Research Institute at Nationwide Children’s Hospital. Dr. Fu’s NIH and privately funded research program focuses on gene therapy, mechanisms of neuropathology and biomarker development targeting MPS III/lysosomal storage diseases and neurodegenerative diseases.

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  • Female

Languages Spoken:

  • English

Research Interests

Research Center:

Areas of Interest:

  • The major focus of Dr. Fu’s research group is to develop efficient gene therapy, using recombinant adeno-associated virus (rAAV) vectors, to treat the neurological and somatic disorders Mucopolysaccharidoses (MPS) in patients. To date, her group has developed two rAAV9-mediated gene therapy approaches for MPS IIIA and MPS IIIB and they are in the process of moving forward for Phase I/II clinical trials in MPS IIIA and MPS IIIB patients.

    Dr. Fu and her group have recently expanded our MPS gene therapy program to develop similar gene therapy approaches for the treatment of other MPS disorders, including Hurler (MPS IH) and Hunter (MPS II) syndrome. They are also interested in the identification of potential biomarkers for different MPS disorders, which has led to the findings of strong blood-brain molecular links and their biomarker potential in mouse models of MPS IIIA and MPS IIIB, and in patients with Alzheimer’s disease.

    Another aspect of their research is to understand how the lysosomal storage of glycosaminoglycans, which is the primary consequence of specific enzyme deficiency, triggers complex secondary pathological changes in MPS disorders, especially neuroinflammation, metabolic impairments and neurodegenration. These may enable us to better understand the disease mechanisms of MPS disorders and identify potential novel therapeutic targets for the therapeutic development to treat lysosomal storage diseases.

Education and Training

Medical School

  • Loude College, Shandong Med. University
    Date Completed: 06/30/1979

Graduate School

  • Chinese Acad. Prevent. Med.
    Date Completed: 06/30/1988

Post Doctoral

  • Institute for Animal Health
    Date Completed: 06/30/1995

Professional Experience


  • Assistant Professor, Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Department of Pediatrics, The Ohio State University, Columbus, Ohio


  • Research Assistant Professor, Division of Genetics and Metabolism, Department of Pediatrics, University of North Carolina at Chapel Hill, North Carolina


  • Postdoctoral Research Associate in Microbiology and Gene Therapy, Division of Genetics and Metabolism, Department of Pediatrics, University of North Carolina at Chapel Hill, North Carolina


  • Postdoctoral Research Associate in Molecular Biology, Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, North Carolina


  • Visiting Scholar in Microbiology, University of Surrey, UK


  • Research Associate in Microbiology, Institute of Microbiology and Epidemiology, Chinese Academy of Preventive Medicine, Beijing, PR China


  • Resident and Instructor in Dept. Infectious Diseases and Epidemiology, Loude College, Shandong Medical University, Shandong, PR China


  • D.M. McCarty, J. DiRosario, K. Gulaid, J. Muenzer, H. Fu. 2009. Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice.  Gene Therapy. Vol. 16, no. 11. (November): 1340-1352.
  • J. DiRosario, E. Divers, C. Wang, A. Charrier, P. Jukkola, J. Etter, H. Auer, V. Best, D.L. Newsom, D.M. McCarty, H. Fu. 2009. Innate and adaptive immune activation in the brain of MPS IIIB mouse model.  J Neurosci Res..
  • H. Fu, L. Kang, J.S. Jennings, S.S. Moy, A. Perez, J. DiRosario, D.M. McCarty, J. Muenzer. 2007. Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult Mucopolysaccharidosis IIIB mice.  Gene Therapy. Vol. 14, no. January: 1065-1077.
  • D.M. McCarty, H. Fu, P.E. Monahan, C.E. Tolson, P. Naik, R.J. Samulski 10(26):2112-8. 2003. Adeno-associated Virus Terminal Repeat (TR) Mutant Generates Self-complementary Vectors to Overcome Rate-Limiting Step to Transduction In Vivo.  Gene Therapy. Vol. 10, no. January: 2112-2118.
  • H. Fu, J. Muenzer, R.J. Samulski, G. Breese, J. Sifford, X. Zeng, D.M. McCarty. 2003. Self-Complementary Adeno-Associated Virus Serotype 2 Vector: Global Distribution and Broad Dispersion of AAV-Mediated Transgene Expression in Mouse Brain.  Mol. Therapy. Vol. 8, no. January: 911-917.
  • H. Fu, R.J. Samulski, T.J. McCown, J. Picornell, D. Fletcher, J. Muenzer. 2002. Neurological correction of lysosomal storage in mucopolysaccharidosis IIIB knock-out mouse model by adeno-associated virus-mediated gene delivery.  Mol. Ther. Vol. 5, no. January: 42-49.
  • H. Fu, C.J. Leake, P.P. Mertens, P.S. Mellor. 1999. The barriers to bluetongue virus infection, dissemination and transmission in the vector, Culicoides variipennis (Diptera:Ceratopogonidae).  Arch. Virol. Vol. 144, no. January: 747-761.
  • Z.T. Kelleher, H. Fu, E. Livanos, B. Wendelburg, S. Gulino, J.M. Vos. 1998. First-generation of mouse artificial episomal chromosomes for shuttling 100 kb of self-replicating human DNA.  Nature Biotechnology. Vol. 16, no. January: 762-768.
  • L.A. Martin, A.J. Meyer, R.S. O’Hara, H. Fu, P.S. Mellor, N.J. Knowles, P.P. Mertens. 1998. Phylogenetic analysis of African horse sickness virus segment 10: sequence variation, virulence characteristics and cell exit.  Arch. Virol-Supplementum. Vol. 14, no. January: 281-293.
  • P.P. Mertens, J.N. Burrough, A. Walton, M.P. Wellby, H. Fu, R.S. O'Hara, S.M. Brookes, P.S. Mellor. 1996. Enhanced infectivity of modified bluetongue virus particles for two insect cell lines and for two Culicoides vector species.  Virology.. Vol. 217, no. 2. (January): 285-293.
  • H. Fu, R. Cai, M. Jia. 1991. The distribution of serotypes and biotypes of Campylobacter jejuni/coli isolated from ten provinces/cities in China.  Chinese J Epidemiol.
  • H. Fu. 1987. Campylobacter and Guillain-Barr syndrome.
Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000