2016 Fall Symposium


Haiyan Fu, PhD
Gene Therapy Targeting the Root Cause
Treatment of Neuropathic Lysosomal Storage Diseases

Haiyan Fu, PhD


Learning Outcomes:
  • Explain the current status of advancement in gene therapy research
  • Evaluate the evolving therapeutic landscape for lysosomal storage diseases
  • Identify candidate diseases and gene delivery approaches for gene therapy

Haiyan Fu, PhD, is a principal investigator at the Center for Gene Therapy, the Research Institute at Nationwide Children’s Hospital. She did her PhD training in virology in the Institute for Animal Health, UK and postdoc training in the laboratory of Dr. Joseph Muenzer, in the University of North Carolina at Chapel Hill. She initiated her own lab at the Center for Gene Therapy, the Research Institute at Nationwide Children’s Hospital in 2004. Her major research focus has been on the development of gene therapy approaches for neuropathic lysosomal storage diseases. Her work have been supported by multiple patient foundations and grants from the NIH, leading to recent IND approvals by the FDA for two Phase 1/2 gene therapy clinical trials in patients with MPS IIIA and MPS IIIB. Her research interests also include better understanding the neuropathology mechanisms and identifying biomarkers, especially neurological biomarkers for neuropathic lysosomal storage diseases.