Dao Pan, PhD

I’m a geneticist with a passion for developing new therapeutic interventions that integrate original discoveries in developmental biology and disease pathophysiology with cutting-edge biotechnology. In my lab, we are particularly interested in rare inherited disorders. Our main research focus is translational medicine to advance scientific insights and treatment options for the defects in the central nervous system (CNS), the organ most resistant to therapeutic interventions due to the relative impermeability of the blood-brain barrier (BBB).

I obtained my PhD in Molecular, Cellular and Developmental Biology & Genetics at the University of Minnesota, where I gained broad experience in neuronopathic genetic disorders such as neurological lysosomal storage diseases (nLSD), stem cell gene transfer, viral vector-mediated gene therapy and preclinical studies in animal models. My subsequent work in a Phase I/II clinical trial of lymphocyte gene therapy and serving as a core director and a co-investigator for National Institutes of Health (NIH) Program Project Grants further fortified my belief and continuous pursuits in developing novel treatment approaches by combining translational and basic research.

Pan Lab research comprises three main areas of discovery. The first is to understand the landscape and roles of neuroinflammation and the remodeling of neurovascular units (e.g., BBB) in the brain pathogenesis of nLSD. The second is to identify potential new therapeutic targets or disease-modifier gene(s) that could influence disease progression. The third is to develop new strategies to improve CNS efficacy and reduce side effects/toxicity in viral vector-mediated stem cell and gene therapies.

Some of the notable discoveries my team and I have made include:

• Demonstrating for the first time that adult hematopoietic and mesenchymal stem cells could be genetically modified by lentiviral vectors in their natural “niche” without any preconditioning, providing a new approach applicable for disease treatment (such as in Hemophilia) and investigation of adult stem cell plasticity
• Identifying two peptide sequence(s) that could facilitate transcytosis of large protein across the BBB and lead to wide-spread protein distribution with improved CNS benefits in a mouse model of Hurler syndrome
• Revealing a new concept that maturing red blood cells, as well as megakaryocyte/platelets, could serve as unconventional and potent in vivo depots for efficient production, protected delivery and effective distribution of lysosomal enzymes
• Discovering that microRNA-143 contributes to the down-regulation of M6P receptor on mature BBB and that the loss of miR-143 in a diseased mouse model led to advanced CNS benefits with wide-spread distribution of enzymes delivered through the peripheral blood, revealing a new target to improve brain drug delivery 

My research team has been continuously funded by the National Institutes of Health (NIH), industrial sponsors, societies and seed initiatives from Cincinnati Children’s and the University of Cincinnati. I am actively involved in committees of regional, national and international scientific societies. I’m on the editorial board of several scientific journals and serve as an academic editor for Advances in Cell and Gene Therapy. I also serve on NIH study sections and several international grant review agencies.

I began working at Cincinnati Children’s in 2003. I have over 23 years of experience in genetics, neuroscience, biotechnology and preclinical studies. I believe deeply in the golden bond between education and the future of science. My lab has supported trainees at all levels, from undergraduate students to postdoctoral researchers with various ethnic backgrounds. We always seek hard-working people with curious minds to join our team.