How to Treat Cystic Fibrosis With Gene Therapy
In 1989, researchers discovered CFTR, the gene that causes cystic fibrosis (CF). Gene therapy is still experimental, but scientists hope that this research will provide answers about treating the cause of cystic fibrosis rather than the symptoms. Gene therapy research has focused on lung cells because respiratory failure accounts for most cystic fibrosis deaths, but scientists hope to be able to use what they've learned to treat all affected genes.
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Instructions
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Use Gene Therapy to Treat Cystic Fibrosis
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Learn about the Cystic Fibrosis Transmembrane Regulator (CFTR) gene that causes CF. To develop the disease, one defective gene must be inherited from each parent.
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Learn that in healthy people, the CFTR protein is found in the lining of major organs and controls the transport of chloride, salt and water across cell membranes. Salt dysregulation in CF patients results in a buildup of excess mucus that promotes bacterial growth, blocks glands and prohibits the movement of digestive enzymes.
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Study the treatment of cystic fibrosis with experimental gene therapy by inserting copies of normal genes into organ linings. While gene therapy is still experimental, it is currently possible to treat cystic fibrosis symptoms, such as bacterial infections, respiratory ailments and pulmonary difficulty, with gene replacement through lung and heart transplantation.
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Replace both lungs in people with severely limited lung capacity caused by cystic fibrosis. Lung transplantation is rare because the risks are so great, but successful transplants can add years to people's lives.
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Do not expect to be cured through a transplant. Lung transplantation does not cure cystic fibrosis because CF genes in other organs are not replaced.
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Study developments in gene therapy research that explore ways to deliver normal genetic material to CF cells in respiratory-tract linings. Researchers are also searching for a way to modify the CFTR protein so that it can properly regulate the transport of chloride between cells.
Combine Therapies to Treat Cystic Fibrosis Symptoms
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Treat cystic fibrosis by clearing lung infections with antibiotics and mucus-thinning drugs.
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Reduce inflammation of lung tissue through the use of anti-inflammatory drugs, such as pain relievers.
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Improve food absorption and weight gain in CF patients through the use of digestive enzymes.
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Clear lung congestion through bronchial-airway-drainage techniques, including Chest Physical Therapy (CPT).
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Maintain the health of CF patients through regular exercise.
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Tips & Warnings
Consult a genetic counselor if you and your spouse are carriers of the CF gene. A child born to parents who are both carriers has a 25-percent chance of developing CF.
