If a person is born with a hereditary disease, can a corrective therapy by taken? Explain with an example.
Person born with hereditary disease can be cured by gene therapy.
The 1st clinical gene therapy was given in 1990 to a 4yr old girl with ADA deficiency. (adenosine deaminase).This enzyme is very important for the immune system to function. This disorder is caused due to deletion of gene for adenosine deaminase.
In some children it can be cured by Bone marrow transplantation, while in others in can be treated by enzyme replacement therapy, in which functional ADA is injected to the patient. But both the treatment is not completely curative.
In gene therapy, lymphocytes from the blood of the patient are grown in a culture outside the body. A functional ADA cDNA is then introduced into these lymphocytes, which are then introduced onto the patients. However, these cells are not immortal and has to be infused periodically. But, if the gene isolate from arrow cells producing ADA is introduced into cells at early embryonic stages , it could be a permanent cure.
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