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Cystic Fibrosis and Gene Therapy

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Cystic Fibrosis (CF), a life-threatening condition that has no treatment, can potentially be cured by gene therapy. The information that I have stated in this essay is used from different articles of ... National Centre for Biotechnology Information, known as NCBI. Gene Therapy can provide us cure for CF in near future. Mutations in cystic fibrosis transmembrane conductance regulator (CFTR) gene results in CF, which is a hereditary disease that causes shortening of lifespan. More than 30,000 people in the United States have cystic fibrosis with more than 1,000 new cases each year. There is a test for CF that measures sweat electrolyte levels as patients have higher concentrations of chlorine and sodium. CFTR gene encodes a protein that is involved in regulation of bicarbonate transport and epithelial sodium channel. People who have CF may have irregular function of CFTR protein. A Major outcome of such mutations causes obstruction by viscous, thick secretions in the gastrointestinal, reproductive, and respiratory tracts. Patients with CF have respiratory epithelia that have unusually high rates of sodium absorption, which impairs mucus transport due to dehydration of airway surface liquid. This causes mucociliary clearance (MCC) mechanisms to fail to remove inhaled bacteria, infecting lower airways and leading to inflammation. DNA and elastase, inflammatory cell contents, increase the viscosity of mucus and contribute in breaking down of tissue [Show More]

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