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Question

When gene targetting involving gene amplification is attempted in an individual's tissue to treat disease, it is known as :

A
Safety testing
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B
Biopiracy
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C
Gene therapy
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D
Molecular diagnosis
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Solution

The correct option is C Gene therapy
Gene therapy is a collection of methods that allows correction of a gene defect that has been diagnosed in a child/embryo.
It involves the correction of a genetic defects by the insertion of functional genes into a person’s cells and tissues to compensate for the non-functional gene.
For example, adenosine deaminase (ADA) deficiency is a disorder caused due to the deletion of the gene for adenosine deaminase which is a crucial enzyme for the immune system to function.
It can be treated by gene therapy where the lymphocytes from the patient's blood are grown in a culture outside the body. A functional ADA cDNA (using a retroviral vector) is then introduced into these lymphocytes and subsequently returned to the patient. However, as these cells are not immortal, the patient will require periodic infusion of such genetically engineered lymphocytes.

Safety testing involves transgenic animals that carry genes which make them more sensitive to toxic substances than non-transgenic animals. The animals are then exposed to toxic substances to study their effects.

Biopiracy refers to the use of bio-resources by multinational companies and other organisations without proper authorisation from the countries and people concerned without compensatory payment.

Molecular diagnosis is the process of of identifying a disease by studying biomolecules, such as proteins, DNA, and RNA, in a tissue or fluid.

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