NPKUA FUNDED RESEARCH

The NPKUA is pleased to highlight the 2017 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 eight 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 2017
Research Awards. 

Click here for a list of the 2016
Research Awards. 

Click here for a list of the 2015
Research Awards.

Click here for a list of the 2014
Research Awards.

Click here for a list of the 2013
Research Awards.

Click here for a list of the 2012
Research Awards.

Click here for a list of the 2011
Research Awards.

Click here for a list of the 2010
Research Awards.

2017 Research Awards

Dr. Katherine Durrer-Deming at the University of North Texas is continuing her research into the ability of a genetically engineered probiotic to lower blood Phe levels in PKU mice.

Dr. Roberto Gramignoli at the Karolinska Institutet in Stolkholm, Sweden is focusing in cell-based therapies to correct metabolic defects.

Dr. Cary O. Harding at the Oregon Health & Science University, is continueing to research gene therapy as a promising approach to cure PKU.

Dr. Eileen K. Jaffe at the Fox Chase Cancer Center is studying how structure changes in the phenylalanine hydroxylase enzyme (PAH) ensure the control of phenylalanine (Phe) concentration.

Dr. Robert Nicholls is Professor of Pediatrics in the Division of Medical Genetics at Children’s Hospital of Pittsburgh of UPMC. Last year, NPKUA funding created the first PKU pig animal model using ge-nome editing. This year Dr. Nicholls and his colleagues will breed pigs that are genetic PKU carriers and ones that have PKU to establish breeding stock and obtain sufficient offspring for experimental and control groups of animals for in depth studies.

Dr. Francjan van Spronsen, Professor of Pediatrics at the University of Groningen, Netherlands, will focus on finding the difference towards personalized PKU treatment.

Dr. Susan Waisbren at Boston Children’s Hospital, will use new techniques to potentially close one of the most important gaps in the knowledge of PKU, namely to define how PKU affects the brain.

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.

Click here for full summaries of the 2017 research projects.

 


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