The mission of the Y.T. and Alice Chen Pediatric Genetics and Genomics Research Center is to develop novel and effective treatments and establish a better understanding of the pathophysiology and long term complications of patients affected with genetic/metabolic disorders. The primary focus of the research will be single gene disorders, including a clinical/translational core team to facilitate implementation of this goal. The new treatment strategies will include, but not be limited to, enzyme replacement therapy, small molecule therapy, stem cell therapy, SiRNA therapy, gene editing, gene therapy and combination therapy approaches.

• Establish a comprehensive clinical/translational research team for the investigation of genetic/metabolic diseases to enhance our understanding of the disease, develop new treatments and demonstrate the safety and efficacy of the treatments.
• Provide the primary resources and optimal environment necessary to stimulate the development of clinical/translational research in genetic/metabolic disorders for the scientists in the Division of Medical Genetics and collaborators throughout the medical center.
• Establish an arm’s length collaboration with the pharmaceutical industry to develop new treatments, demonstrate effectiveness, and investigate beyond industry marketing interests.

To accomplish its mission, the Chen Center relies on proceeds and royalties from previous discoveries; federal, industry and foundation grants; and generous contributions from Alice and Y.T. Chen. Many individuals, including grateful patients and families, also make philanthropic contributions to support the research.