explain how gene therapy has been used to treat cystic fibrosis. explain why gene therapy has not been used to cure cystic fibrosis.
explain how gene therapy may be used to treat haemophilia and cystic fibrosis.
thank you
how gene therapy might be used to treat cystic fibrosis
How would gene therapy treat these two diseases. Cystic fibrosis and parkinson disease???? I’m looking for information that will describe the treatment using an animal model for testing… but i’m willing to take any information.
The primary aim of medical and scientific research on cystic fibrosis is to understand, treat and cure cystic fibrosis. The research also focuses on serious symptoms and complications of the disease.
Cystic fibrosis research on gene therapy and protein therapy has made new developments. Gene therapy using liposomes is the technique of adding a healthy copy of the gene. Protein therapy is used to repair the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Drugs for replacing the function of CFTR protein have been developed. Research groups are engaged in developing new drugs to move this protein to the membrane.
Cystic fibrosis research has spread to the area of improving symptom control which includes controlling lung infections, reducing inflammation and improving nutrition.
Another fertile area of interest is regarding the cause of cystic fibrosis. The research on genetic disorders explains the factors that regulate channel activity and their effect on the plasma membrane. This research proves that the inherited disorders of cystic fibrosis include chronic pulmonary disease and hypertension. Research done on the regulation of gene expression identifies the factors that control gene expression. The study is conducted on the effect of alterations in these factors. Researchers have developed optical imaging aids for cystic fibrosis study. Optical sensing tools are used to investigate adenosine triposphate release and its role in cystic fibrosis.
Many researches are conducted to improve the treatment of cystic fibrosis. New medicines have been invented to remove the secretions in the lungs. The removal of secretions can be measured by inhaling a small amount of radioactive gas. New methods are developed to assess the deposition and clearance of the radioactive gas. Cystic fibrosis research has proved that the effect of physiotherapy is not uniform in all patients. Physiotherapy made a positive effect in majority of patients. But some patients experience deterioration in ventilation. Researchers on this disease investigate the disordered breathing and the effect of exercise on the patients. Arm exercises result in overexpansion of lungs and this may cause breathlessness in severe cystic fibrosis patients.
By: Kent Pinkerton
About the Author:
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Cystic fibrosis is a medical condition that starts in early childhood, sometimes as early as from the time of birth. A defective gene causes the problem and there is no known cure, though there are some promising experiments going on towards gene therapy. The medical profession understands the symptomatic treatment well and the average lifespan of persons with cystic fibrosis is increasing.
Nature’s mechanism for filtering out the dust and microorganisms people breathe in is to secrete thin mucus in the airways and the lungs, and clear it through the nose or the digestive tract. For the person with cystic fibrosis, this mucus, which normally is thin and slippery, becomes thick and sticky. Other affected and thickened secretions are sweat, digestive juices and the reproductive system.
Since the lungs are congested, the pulmonary capacity drops. The blocked airways cause severe breathing difficulties and asthma-like wheezing. The digestive juices from the pancreas and liver do not reach the intestine, as the ducts get blocked. The fats and proteins are not digested. Though the patient eats normally, maybe even in excess, he is undernourished and is under weight. Fat-soluble vitamins become deficient.
There are approximately ten million symptomless carriers of the defective cystic fibrosis gene in America. A person needs to inherit two defective cystic fibrosis genes to be afflicted with cystic fibrosis. Every time two carriers produce a child, the chances are 25% that the child may be affected with cystic fibrosis, 50% that the child may be a carrier and 25% that the child may be a non-carrier.
To diagnose cystic fibrosis, the laboratory carries out a sweat test. The treatment is only symptomatic. We cannot at this stage make the secretions thin, but can neutralize the effects of thickened secretions by regular treatment and management. A high-fat diet with enzyme and vitamin supplements helps the patient with nutrition.
A strict lifestyle regime to prevent complications is very important. Drinking plenty of liquid loosens the mucus. Regular exercise, as much as possible, keeps the respiratory system clear and improves the cardiovascular system. The cystic fibrosis patient must avoid smoky or dusty places. Hand cleaning is a simple but very effective step to prevent infection.
Few cystic fibrosis patients lived beyond their teens in the past, but thanks to improved management, 40% of the 30,000 Americans with cystic fibrosis are over 18 and many are into their 30s and 40s.
By: Kent Pinkerton
About the Author:
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i want to know how gene therapy can help all types of cystic fibrosis.and i also want to know what are some of the really bad cystic fibrosis diseases or diseases that are alike them.
