The major goals of our laboratory are to comprehensively understand the biology of recombinant AAV vectors and the vector-host interactions and to develop new AAV vector-mediated gene and cell therapies to treat various human diseases. To achieve these goals, we take multi-disciplinary approaches in which we use molecular, cellular and structural biology techniques, bioinformatics, computational biology, computer simulation, various high-throughput technologies including DNA and RNA barcoding, the next-generation sequencing (NGS), and mass spectrometry. We study AAV in test tubes, in tissue culture cells, and in animals of various species, both small and large animals. Our laboratory is a part of the Oregon National Primate Research Center (ONPRC) and has been productively conducting AAV vector research using non-human primates. In addition, our high performance computing projects have been supported by the Pittsburgh Supercomputing Center for many years. 

Hiroyuki Nakai
Hiroyuki Nakai, M.D., Ph.D.