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A GM pig that gets cystic fibrosis is helping scientists understand the causes of the disease. The pig strain can also be used to test new therapies for CF
While the gene mutation that causes cystic fibrosis has been known for over 20 years, it is still unclear exactly how this mutation results in the condition.
In humans, thick sticky mucus accumulates in the lungs and digestive tract. Patients have difficulty breathing and digesting food and suffer frequent lung infections. The CF gene mutation affects the production of a protein called cystic fibrosis transmembrane conductance regulator (CFTR). CFTR forms a channel in cells that helps maintain salt and water balance in cell membranes.
Researchers discovered that in the GM pig most of the CFTR protein is misprocessed. Only a small amount of the protein gets to the cell membrane where it is needed. If researchers can understand why it is misprocessed then they could direct treatments to transport more of the protein to the cell membrane.
The GM pig has similar symptoms to CF in humans and the CFTR protein behaves in the same way as in humans. Researchers hope to use the model to test new treatments for cystic fibrosis.
Read more about cystic fibrosis and animal research here.
Last edited: 11 January 2022 10:59
