Sanford Children’s Health Research Center

“Junk DNA” drives embryonic development

“Junk DNA” drives embryonic development

Mark Mercola, Ph.D., and his team discovered that microRNAs play an important role in the allocation of cells into three germ layers—ectoderm, mesoderm, and endoderm—during development that give rise to all tissues and organs in the body.

Exciting clinical trial news for children with inherited bone disease

Exciting clinical trial news for children with inherited bone disease

José Luis Millán, Ph.D., has studied hypophosphatasia, an inherited disease that makes bones fragile, for the past 15 years. In a recent clinical trial, patients were treated with a new enzyme replacement therapy, showing significant improvement.

Rare bone disorder reveals new insights into autism

Rare bone disorder reveals new insights into autism

Researchers at Sanford-Burnham recently used a mouse model to investigate human multiple hereditary exostoses. They foundt symptoms that meet the three defining characteristics of autism: social impairment, language deficits, and repetitive behavior.

About the Sanford Children's Health Research Center

I have the utmost confidence that this collaboration will promote solutions to some of the most troubling health issues that affect children.

- Denny Sanford

Naming donor and Honorary Trustee

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.

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 Sanford-Burnham’s extremely powerful core facilities that give flight to bold new ideas and extend imagination into tangible clinical realities.

Recent Developments

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