NPKUA FUNDED RESEARCH

The NPKUA is pleased to highlight the 2016 Research Awards. These awards are made possible by our member organizations who raise funds each year on the local level for research. Thank you for helping make these awards possible as we work towards improving treatment options for PKU and accelerating the timeline for a cure.

The overall funding strategy of the NPKUA is to support projects that will promote advances in the treatment and management of PKU, with the long term goal of facilitating the development of a cure. The NPKUA has a Scientific Advisory Board made up of eminently qualified doctors, researchers and clinicians that evaluate the proposals and provide a recommendation to the NPKUA Board each year for funding.

Impact of Funding

The last seven years have seen great strides in finding ways to improve treatment for PKU. We are pleased to share our annually funded research awards that focus on new and innovative treatment options for PKU and accelerating the timeline for a cure.

Click here for a list of the 2010
Research Awards.

Click here for a list of the 2011
Research Awards.

Click here for a list of the 2012
Research Awards.

Click here for a list of the 2013
Research Awards.

Click here for a list of the 2014
Research Awards.

Click here for a list of the 2015
Research Awards.

Click here for a list of the 2016
Research Awards. 

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.

 


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