1) Gene therapy is a novel approach to treating cancer currently in clinical tri
ID: 68733 • Letter: 1
Question
1) Gene therapy is a novel approach to treating cancer currently in clinical trials. But, it is not without risk. Why do you think someone would volunteer for this type of a study? (Explain your reasoning)
2) Why would someone opt to not seek treatment? (Explain your reasoning)
3) Do you think that a survival rate of 2 out of 17 patients can be counted as a success? Explain why or why not?
4) What is the mimimum survival rate required to claim that a treatment is efficacious? Explain your opinion.
5) Would you consider this sort of treatment if you were faced with an inoperable cancer that has not responded to other conventional treatments? Explain why or why not.
Explanation / Answer
1). Gene therapy is an experimental approach to treat the disease by inserting therapeutic genes or changing gene expression levels in the patient’s cells. Genes are nucleic acid polymers; these therapeutic genes used in gene therapy generally encode a functional gene that replaces mutated gene. Potential treatment for single gene genetic disorders such as haemophilia, muscular dystrophy, cystic fibrosis, etc. was demonstrated. Treatment of cancer, AIDS (acquired immune deficiency syndrome), Alzheimer’s disease and rheumatoid arthritis are also under active investigation. Approaches of gene therapy include the following (Sanjukta misra, 2013).
Before starting gene therapy, identification of defective genes using DNA probe and availability of normal genes is important. Two types of gene therapy are somatic gene therapy and germ line gene therapy.
Clinical trials in gene therapy is a very critical phase to pass through. One can participate as a volunteer in gene therapy if they are explained with all the possible effects of the therapy with well proven positive results in pre-clinical trials. However, great concern about the volunteer in several aspects must be implemented and must assure almost 0% adverse effects.
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