Skeletal Disease in a Dish: Using novel in vitro disease models produced from patient induced pluripotent stem cells to reveal pathogenic mechanisms and explore treatments for genetic skeletal disorders

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Inherited skeletal disorders are a significant disease burden. Many gene mutations have been defined but we only have limited understanding about how they cause the disease. We will use patient skin cells and a new in vitro cell reprogramming technology to induce them to form cartilage and bone cells to produce mutation-specific “disease in a dish” models. These models will allow us to answer questions about how specific mutations cause disease and test novel drug therapies