Cystic Fibrosis: A Case For Gene Therapy

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Cystic Fibrosis "“ A Case For Gene TherapyCystic Fibrosis is caused by the mutation in the gene, which is located in the long arm of chromosome 7, causing the loss of a single amino acid from a large protein and thus disrupting the binding site for ATP in the protein. Cystic Fibrosis is inherited genetically from one generation to the next due to an autosomal recessive allele. People suffering from Cystic Fibrosis are homozygous for the gene causing the disease, which means that both parents must be carriers of the disease. For these parents, they have one in four chances to give birth a Cystic Fibrosis sufferer.

The cells in the lung, sweat glands, intestine, and pancreas of a Cystic Fibrosis sufferer fails to get rid of chloride ions. Normally, these leave the membrane of the cells through ion channels. In sufferers of Cystic Fibrosis, these channels are blocked.

Thick mucus builds up in the lungs, providing rich breeding location for bacteria and other microorganisms. Mucus are thick and unable to flow, causing problems in the lungs that become congested with mucus. As a result, the lungs become prone to infection, disabling them to walk far or to do rigorous activities without stopping for a long cough. The pancreas of the sufferer also becomes blocked and the protective lining of the intestine becomes defective, preventing food to be digested completely.

Presently, the most common method to help the patients with the disease is by doing daily physiotherapy for their lung problems, and a periodic treatment with pancreatic extract. In young children, parents play an essential role in this kind of treatment. Some others may be lucky enough and have a heart-lung transplant to deal with the complications of lung disease. However, it is now possible to diagnose cystic...

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