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What is the gene therapy for cystic fibrosis?

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in particular. Gene therapy typically aims to supplement a defective mutant allele with a functional one. Although the technology

is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is often lumped together with them.

Cystic fibrosis ultimately could be cured if safe and effective methods could be found to replace the defective CFTR gene with an intact gene in affected tissues. This process is called gene therapy. In the case of cystic fibrosis, gene therapy involves inhaling a spray that delivers normal DNA to the lungs. The goal is to replace the defective cystic fibrosis gene in the lungs to cure cystic fibrosis or slow the progression of the disease. During such a treatment, shuttle vehicles called vectors deliver a functional copy of the defective gene-in this case, CFTR-either to cells throughout the body or to specific affected tissues such as the lungs. These vectors most commonly are derived from viruses that can infect the target cells, although non-virus-based vectors also are available. Once the new CFTR gene has entered the cell, the cell's biochemical machinery must recognize it and use it as a template for the production of functional protein.

More information on cystic fibrosis

What is cystic fibrosis? - Cystic fibrosis is a hereditary disease of the exocrine glands. Cystic fibrosis affects the pancreas and the glands that secrete sweat and mucus.
What causes cystic fibrosis? - Cystic fibrosis is caused by a defect in the person's genes. The defective gene that is responsible for causing cystic fibrosis is on chromosome 7.
What're the complications of cystic fibrosis? - Most people with cystic fibrosis also have digestive problems. People with cystic fibrosis have an increased risk of lung infections.
What're the symptoms of cystic fibrosis? - The symptoms of cystic fibrosis usually develop during early childhood. Cystic fibrosis is characterized by the production of very thick and sticky mucus.
How is cystic fibrosis diagnosed? - Family history, persistent respiratory disease, or clinical evidence of pancreatic insufficiency may suggest the diagnosis of cystic fibrosis.
What's the treatment for cystic fibrosis? - Treatment for cystic fibrosis consists of the intake of digestion enzymes, nutritional supplements, percussion and postural drainage of the lungs, improved antibiotics.
What cystic fibrosis medications are available? - Cystic fibrosis medications include H2 receptor antagonists, oral corticosteroids, ibuprofen, erythromycin, amoxicillin-clavulanate, etc.
What cystic fibrosis diet is suggested? - People with cystic fibrosis usually require high-calorie diets and vitamin supplements. Most people with cystic fibrosis need to take pancreatic enzymes.
What's the gene therapy for cystic fibrosis? - Gene therapy for cystic fibrosis typically aims to supplement a defective mutant allele with a functional one.
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