Cystic fibrosis (CF) is a common autosomal recessive disease caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR). CF affects multiple organs, including lungs, pancreas, intestine, liver, sweat glands, gallbladder and the male genital tract. Airway infection and inflammation currently cause most of the morbidity and mortality. Although several therapies have improved the lives of patients, current treatments are inadequate and CF remains a lethal disease. Our knowledge about the pathogenesis of the disease, its progression, and the state of the neonatal lung is inadequate. These gaps in our knowledge have hindered attempts to develop better treatments and preventions for CF lung disease. A major impediment to addressing these issues had been limitations of animal models. We generated CF pigs that replicate many of the key features of human CF disease including intestinal obstruction, exocrine pancreatic destruction, micro-gallbladder, vas deferens abnormalities, focal biliary cirrhosis, congenital airway structural abnormalities, and airway and sinus infection with time. The goals of the Animal Models Core will be to (1): Provide Program investigators with non-CF and CF pigs so that they can successfully complete their project aims; (2) Build new genetically engineered CF pig models; (3) Assist projects in the harvesting of biologic samples from live animals (e.g., bronchoalveolar lavage fluid and blood), the care and analysis of study animals, record keeping, and coordinate animal usage among projects. The Animal Models Core will function seamlessly through already established interactions with the Project Leaders and Core Directors. The success of the Animal Models Core is ensured because of the commitment, experience, and expertise that the personnel bring to the Core.
CORE A ? ANIMAL MODELS PROJECT NARRATIVE Cystic fibrosis is a common life-shortening genetic disease that causes progressive lung failure due to recurrent infections and chronic inflammation. These studies will use cystic fibrosis pigs to better understand the early pathogenesis of CF.
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