Gene therapy is generally defined as introducing a new gene (or DNA sequence) or a “repaired” version of an existing defective gene into a living human cell for therapeutic benefits. Since the first gene therapy clinical trial initiated in 1991, this new therapy has evolved together with new developments in biotechnologies and better understanding of human diseases. There are currently more than 1,400 gene therapy clinical trials worldwide for treatment of diseases such as cancers, genetic diseases and infectious diseases. However, the actual blooming of this therapeutic approach for widespread clinical applications is still waiting for more efforts and improvements on basic research of gene transfer and cell targeting techniques, vector development, stem cell technology, and the biology of targeted cells, etc., as well as on issues encountered when applying preclinical work into actual treatment for patients.
Dao Pan at Cincinnati Children's Hospital Medical Center has long-term research interests in combining translational and basic research on virus-mediated gene transfer and disease treatment for lysosomal storage disease (especially mucopolysaccharidoses, MPS) disorders, which are often associated with both systemic and central nerve system (CNS) abnormalities, as well as early childhood death.