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Question
The given picture depicts the steps involved in gene therapy. Select the disorder/s for the treatment of which this technique has been successfully applied.
The given picture depicts the steps involved in gene therapy. Select the disorder/s for the treatment of which this technique has been successfully applied.
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Solution
The correct option is C Both (A) and (B)
The disease is caused by a mutation in a gene on chromosome 20. The gene codes for the enzyme adenosine deaminase (ADA). Without this enzyme, the body is unable to break down a toxic substance called deoxyadenosine. The toxin builds up and destroys infection-fighting immune cells called T and B lymphocytes. There are no real cures for ADA deficiency, but doctors are trying to restore ADA levels and improve immune system function with a variety of treatments like gene therapy by inserting synthetic DNA containing a normal ADA gene into immune cells. The normal ADA genes are delivered to immature blood cells isolated from the babies umbilical cords. Tumor necrosis factor alpha (TNF-) is a multifunctional cytokine that is secreted by macrophages and can elicit a variety of effects: it can cause cytolysis of certain tumor cells; TNF is known for its ability to induce haemorrhagic tumor necrosis and tumor regression. Gene therapy targets the expression of therapeutic genes to the desired target cells and makes it effective for cancer treatment.
The disease is caused by a mutation in a gene on chromosome 20. The gene codes for the enzyme adenosine deaminase (ADA). Without this enzyme, the body is unable to break down a toxic substance called deoxyadenosine. The toxin builds up and destroys infection-fighting immune cells called T and B lymphocytes. There are no real cures for ADA deficiency, but doctors are trying to restore ADA levels and improve immune system function with a variety of treatments like gene therapy by inserting synthetic DNA containing a normal ADA gene into immune cells. The normal ADA genes are delivered to immature blood cells isolated from the babies umbilical cords. Tumor necrosis factor alpha (TNF-) is a multifunctional cytokine that is secreted by macrophages and can elicit a variety of effects: it can cause cytolysis of certain tumor cells; TNF is known for its ability to induce haemorrhagic tumor necrosis and tumor regression. Gene therapy targets the expression of therapeutic genes to the desired target cells and makes it effective for cancer treatment.
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