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What Do We Know About Gene Therapy and Cystic Fibrosis?

Started by Epoch-Times-Bot, Oct 08, 2022, 01:00 PM

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Epoch-Times-Bot

Oct 08, 2022, 01:00 PM
What Do We Know About Gene Therapy and Cystic Fibrosis?

Cystic fibrosis (CF) is the most common, fatal genetic disease in the United States. About 30,000 people in the United States have the disease. CF causes the body to produce thick, sticky mucus that clogs the lungs, leads to infection, and blocks the pancreas, which stops digestive enzymes from reaching the intestine where they are required in order to digest food.
Mutations in a single gene—the Cystic Fibrosis Transmembrane Regulator (CFTR) gene—cause CF. The gene was discovered in 1989. Since then, more than 900 mutations of this single gene have been identified.
In normal cells, the CFTR protein acts as a channel that allows cells to release chloride and other ions. But in people with CF, this protein is defective and the cells do not release the chloride. The result is an improper salt balance in the cells and thick, sticky mucus. Researchers are focusing on ways to cure CF by correcting the defective gene or correcting the defective protein....

Source: What Do We Know About Gene Therapy and Cystic Fibrosis?

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