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• W1649091874 abstract "Publisher Summary This chapter focuses on cystic fibrosis (CF) that is the most common genetic disease in North America, with an estimated incidence of one in 2750 live births and a carrier frequency of approximately 4%. Adeno-associated virus vectors are used for gene therapy of CF. CF can affect virtually every organ in which there is epithelial ion transport, the primary clinical manifestations of the disease are caused by exocrine pancreatic insufficiency and obstructive lung disease. Pancreatic disease is readily compensated for in most patients by use of oral pancreatic enzyme supplementation. CF lung disease continues to be the leading cause of morbidity and mortality among CF patients. A fundamental understanding of the normal histology of the airway and the pathology of CF lung disease is essential for a complete appreciation of the CF gene therapy problem." @default.
• W1649091874 created "2016-06-24" @default.
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• W1649091874 date "1998-01-01" @default.
• W1649091874 modified "2023-10-18" @default.
• W1649091874 title "[53] Adeno-associated virus vectors for gene therapy of cystic fibrosis" @default.
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• W1649091874 doi "https://doi.org/10.1016/s0076-6879(98)92055-9" @default.
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