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Type 1 diabetes, formerly called insulin-dependent diabetes, juvenile diabetes or childhood onset diabetes, affects 1 to 2 million people in the U.S. and millions more worldwide. In this country, it is second only to asthma as the most common chronic disease in children. However, it may begin at any age, when for yet-unknown reasons, a person's immune system mistakenly attacks beta cells that produce insulin. Patients with this type of diabetes are dependent for life on insulin injections or insulin medications.

The Children's Hospital of Philadelphia has set for itself the ambitious goal of advancing pediatric care worldwide. Its researchers are following multiple strategies to preventing, treating and possibly even curing type 1 diabetes.

Gene Discovery Helps Reveal Treatment Targets on Diabetes Pathways

Because all types of diabetes are complex diseases, affected by interactions among multiple genes, genomics researchers here focus on identifying all the genes involved, as a foundation for further investigating how genes function on biological pathways that lead to diabetes. At the Children's Hospital's Center for Applied Genomics, the world's largest pediatric genotyping project, a study team in July 2007 identified a previously unknown gene variant that raises a child's risk of type 1 diabetes. Using highly automated DNA scanners, the researchers have since discovered and confirmed other gene variants, and will augment the list with other findings in the coming months. Hakon Hakonarson, M.D., the center's director, says "better understanding of these gene pathways may shed light on ways to intervene early in life with targeted drugs or cell therapies that could prevent diabetes from developing." (Center for Applied Genomics: For more information, visit Welcome to The Children's Hospital of Philadelphia.
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