Gene Therapy And Sickle Cell Anemia Biology Essay


The defective gene is in the hemoglobin. Bone marrow containing the defective hemoglobin gene was removed from the mice and genetically corrected by the addition of the anti-sickling human beta-hemoglobin gene. The corrected marrow was then transplanted into other mice with sickle cell disease. The genetically corrected mice began producing high levels of normal red blood cells and showed an affected decline in sickled cells. Scientists are hopeful that the techniques can be applied to human gene transfer using autologous transplantation, in which some of the patient's own bone marrow cells would be removed and genetically corrected.

Symptoms of (SCA) are abdominal pain, bone pain, breathlessness, delayed growth, fatigue, fever, paleness, rapid heart rate, ulcers on the lower legs (in adolescents and adults), yellowing of the eyes and skin (jaundice), chest pain, excessive thirst, frequent urination, painful and prolonged erection, poor eyesight/blindness, and strokes. SCA is usually inherited from parents who are carriers, who have the sickle cell trait-a milder form of sickle cell anemia, or one abnormal hemoglobin. Patients with sickle cell disease need ongoing treatment, even when they are not having a painful crisis. They should take supplements of folic acid (essential for producing red blood cells) because red blood cells are turned over so quickly.

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The treatment of SCA differs according to the frequency of the disease. The purpose of treatment is to manage and control symptoms, and to limit the frequency of emergencies. During a sickle cell emergency, you may need certain treatments. Painful frequencies are treated with pain medicines and by drinking plenty of fluids. Non-narcotic medications may be effective, but some patients will need large doses of narcotics. Hydroxyurea is a drug some patients use to reduce the number of pain frequencies, including chest pain and difficulty breathing. Antibiotics and vaccines are given to prevent bacterial infections, which are common in children with sickle cell disease.

Blood transfusions are used to treat a sickle cell crisis. They may also be used on a regular basis to help prevent strokes.

The third disease is Cystic Fibrosis (CF). CF is a common recessive genetic disease which affects the entire body, causing progressive disability and often early death. There have been several trials for curing cystic fibrosis, but the most widespread study deals with gene therapy. Adenoviruses, which are like gene carriers, have become on close study since they are able to infect human airways and result in only a minor sickness such as the common cold. The goals of gene therapy in this disease are to design an effective method of introducing the cloned gene into the cells and to make the gene function inside the somatic cell. To improve the function of the gene, the appropriate controlling basics of the gene must be determined, such as guiding a copy in suitable cells. Even though the development of gene therapy is uncompleted, problems have already ascended. The number of cells that must be correct for clinical benefit is unknown and the treatment may lead to over expression which could cause toxicity. The immune system may reject the route. Gene therapy is also surrounded by a plethora of ethical concerns. The questions of when to treat a patient, who to treat, authorization of a minor treatment, and cost effectiveness of the procedure all have a benefit on the future of gene therapy for cystic fibrosis.

Symptoms of CF include:

delayed growth

failure to gain weight normally during childhood

no bowel movements in first 24 to 48 hours of life

salty-tasting skin

nausea and loss of appetite

weight loss

coughing or increased mucus in the sinuses or lungs


nasal congestion caused by nasal swellings

recurrent incidents of pneumonia


increased coughing

increased shortness of breath

Cystic Fibrosis is a genetic disorder. A child must inherit a specific gene from both parents to get cystic fibrosis. An early diagnosis of CF can help the patient in survival. Monitoring is very important.

The average cost of gene therapy is $100,000 per individual. However, the genetic testing is also expensive. Genetic testing may vary from hundreds to thousands. The therapy itself differs according to diseases. Many people cannot afford the payments of gene therapy. Since gene therapy is still experimental people should pay more money for the therapy to receive good results. C:\Documents and Settings\Owner\Local Settings\Temporary Internet Files\Content.IE5\A32993HW\MP910216577[1].jpg

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There are many causes of gene therapy being fatal. Some are that gene therapy isn't professional yet, the expenses, and that it may not cure the patient successfully. I think that gene therapy shouldn't be used more often because it is still experimental and has a very low expectancy of succeeding. It may probably be fatal at some point related to the disease. Many people couldn't afford the expenses of gene therapy, so it is very low to be often done. And the last factor is that scientists haven't yet discovered cures of specific disease or they found the cure but it doesn't treat the disease successfully. People can spend money on the therapy but get no good results.