Genes and Cloning Aug. 24, 2011- Nine years after getting gene therapy for a rar

Question

Genes and Cloning Aug. 24, 2011- Nine years after getting gene therapy for a rare, inherited immune system disorder often called "bubble boy disease," 14 out of 16 children are doing well researchers report. The children were born with severe combined immunodeficiency disease (SCID).. [Nine years after treatment] 14 out of the 16 children had working immune systems and were leading normal lives "These children, who would have died very young without treatment, are participating in life as fully as their brothers and sisters," researcher H. Bobby Gaspar, MD, PhD, tells WebMD. "Most of them are going to school, playing ball, and going to parties."... .. For most of the children, gene therapy was a success. But one boy who had the X1 form of SCID developed treatment-related leukemia. The complication was not unexpected Gaspar says, because four children with the X1 form of SCID in a French study had developed leukemia after getting the gene therapy Was it morally permissible for these researchers to use gene therapy even though they knew that some children might get leukemia after treatment? Should the treatment be used only if there are no risks involved? What is gene therapy was the only possible treatment for the genetic disease, and most children died within two years of being diagnosed? How would these facts change your assessment of the morality of using gene therapy?

Explanation / Answer

In gene therapy, the nucleic acid is delivered therapeutically to the patient in order to treat a disease. The defective genes are replaced by the normal genes which can be used to correct genetic disorders. In the given case, gene therapy is being used to cure SCID in children. These children may have died within two years of the disease being diagnosed.

If gene therapy is the only method to cure such individuals, it can be used to treat these children. However, the effects of the gene therapy need to be thoroughly experimented. Any drug needs to be clinically examined and tested for safety. In this case, the doctors knew that children with X1 developed leukemia after treatment. So, the moral issue is that whether the doctor has decided to do the correct task.

Before the treatment with the gene therapy, the method had to be experimented well upon animals or in the similar conditions which the boy has suffered from. But sometimes, a treatment may be used even if a small risk is involved. In case, gene therapy was the only method to treat these children, it is better to try gene therapy because something is better than nothing and these children would have died within 2 years. These facts change the morality about using gene therapy because 14 out of 16 children were able to survive and lead a normal life. The success rate was high.

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