Targeting therapy to molecular mechanism of disease in obesity and related metabolic disorders

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Obesity and its complications such as diabetes are major and growing public health problems. We have been investigating genetic factors involved in human obesity and a condition called insulin resistance which leads to diabetes, by focusing on patients with severe forms of these diseases from an early age. We have found defects in several genes that cause these diseases. In one disease, called congenital leptin deficiency, we have been able to show the dramatically beneficial effects of leptin therapy. We now want to study the effects of different diets and drug treatments including leptin in additional patient groups. Defining treatment responses in patients with a known genetic condition that effects a key system in the body will help us to understand and treat these patients but also guide our approach to the more common forms of these important diseases.