The research interests of Raymond Hickey, M.S., Ph.D., are in cell and gene therapy for metabolic liver disease. The short-term goals of Dr. Hickey's research are to demonstrate efficacy and safety of ex vivo gene therapy in hepatocytes in small and large models of metabolic liver disease using both gene addition and gene correction strategies.
Since Dr. Hickey's arrival at Mayo Clinic in 2012, he has developed a unique skill set that includes in vivo and ex vivo gene manipulation with viral vectors, stem cell and differentiated cell transplantation, and noninvasive cell imaging using nuclear imaging.
- Developing models of metabolic liver disease. Dr. Hickey and colleagues have pioneered the use of gene targeting and somatic cell nuclear transfer to develop large models of inborn errors of metabolism, including the first genetically engineered model of a liver disease — tyrosinemia type I.
- Ex vivo liver-directed gene therapy. Dr. Hickey and colleagues have demonstrated ex vivo hepatocyte-directed gene therapy with lentiviral vectors and autologous cell transplantation to be an efficacious alternative to whole-organ transplantation in small and large liver disease models.
- Noninvasive imaging of transplanted cells. Dr. Hickey and colleagues have established novel imaging protocols to noninvasively and longitudinally monitor transplanted cells in vivo using clinically relevant nuclear modalities, including single-photon emission computerized tomography and positron emission tomography.
Significance to patient care
The efficacy and safety of ex vivo gene therapy has been clearly demonstrated in the hematopoietic field, in which dozens of patients have recently undergone successful autologous cell and gene therapy using lentiviral vectors. Therefore, successful completion of Dr. Hickey's studies in preclinical models will lead directly to bringing this regenerative therapy to the clinic to treat the multiple metabolic liver disorders that are currently only curable by organ transplantation.