Diseases that predominantly affect children are too often unaddressed by the biopharmaceutical industry and government funding agencies. Rare childhood diseases – those affecting fewer than 200,000 in the U.S – are especially overlooked. The mission of the Sanford Children’s Health Research Center is to generate translational discoveries about such childhood diseases as diabetes, Duchenne muscular dystrophy, hypophosphatasia (bone disorders), and congenital disorders of glycosylation.
What are the goals and activities of the Center?
Our goal is to be a world leader in providing solutions to childhood diseases. Our approach is to combine outstanding basic research with innovative technologies to translate discoveries into clinical benefits.
The Center emphasizes developmental biology and has core strengths in stem cells to help understand what goes wrong in children with inherited diseases and provide insights into ways to regenerate and replace defective organs and tissues.
Team-based problem solving
We employ a multi-disciplinary strategy to assemble complementary talents and focus them on solving problems in childhood health. Our team includes expertise in genetic model organisms for developing new strategies in regenerative biology. We employ chemical biology approaches to identify molecules that modulate drug targets and cellular pathways in therapeutically useful ways, setting the stage for the production of innovative new therapeutics.
The Center’s team is bolstered by having access to SBP's extremely powerful core facilities that give flight to bold new ideas and extend imagination into tangible clinical realities.
We build relationships that will lead our discoveries to the clinic.
Our goals are to license small molecules directed at novel targets to pharmaceutical companies, identifying new disease targets, and discover new diseases. We form collaborations with clinical groups, including Rady Children’s Hospital in San Diego, to take our research from the lab bench to the patient’s bedside.
Human Genetics Program
Technological breakthroughs are helping diagnose and explain known genetic diseases.