2016 Research Awards
Dr. Juan Cabrera-Luque is Research Assistant Professor at George Washington University. His research aims to establish a novel in vitro model to study the effects that high blood phenylalanine concentrations have at the human blood brain barrier level.
Dr. Katherine Durrer-Deming at the University of North Texas is researching the ability of a genetically engineered probiotic to lower blood Phe levels in PKU mice.
Dr. Cary O. Harding at the Oregon Health & Science University, is researching gene therapy is a promising new approach to treating metabolic diseases including phenylketonuria (PKU).
Dr. Roberto Gramignoli at the Karolinska Institutet in Stolkholm, Sweden has described the importance and peculiar characteristics of amnion epithelial cells (AEC) in the human placenta.
Dr. Nicholls is Professor of Pediatrics in the Division of Medical Genetics at Children’s Hospital of Pittsburgh of UPMC. In the first year of NPKUA funding, Dr. Nicholls and colleagues (including Dr. Randall Prather at the University of Missouri) developed reagents for genome editing of PAH in pig embryos to produce a miniature pig model of PKU.
Dr. Dong Yizhou is Assistant Professor at Ohio State University. Dr. Yizhou hypothesizes that gene corrections to PAH will produce a functional PAH protein and recover the metabolic process, resulting in a cure for PKU.
Dr. Paulo Roque Lino at iMed.ULisboa (Research Institute for Medicines, Faculty of Pharmacy, University of Lisbon, Portugal) was awarded a fellowship for the second year to focus on the development of an Enzyme Reposition Therapy approach to PKU.
Click here for full summaries of the 2016 research projects.