Gene Therapy And Cystic Fibrosis

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Cystic fibrosis is an inherited disease that mostly has been seen in the Caucasia population [1] but also was found in different ethnicities. Cystic fibrosis affects some organs, especially those that have epithelial cells. Cystic fibrosis disease mainly affects the respiratory and digestive tracts.[2] Cells that produce mucus, secrete stickier and thicker. This is the main effect of cystic fibrosis disease. While this mucus is stickier and thicker it causes problems in the organs that have a mucus layer surrounding cells.[1] Mainly in the lungs, since the mucus is thicker and stickier, individuals have a problem transporting oxygen to the cells due to the excess amount of mucus on the layer of the lung. That causes patients to have treatment to regulate their blood oxygen levels. A different story happens in the pancreas. Small tubes that transfer the pancreatic digestive enzymes cannot pass through the tubes due to the blockage on the tubes caused by the thicker mucus.[1] Relatively, digestive enzymes cannot pass through, patients have digestive problems and mostly they face malnourishment. Since this life-limiting and potentially fatal disease is hereditary, there is no definitive treatment method yet. However, with the developing technology, gene therapy is thought to be the most promising solution for cystic fibrosis patients.