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Adenosine deaminase (ADA) deficiency is ...

Adenosine deaminase (ADA) deficiency is a hereditary disease, where ADA, which is crucial for functioning of immune system is absent. Ex plain how ADA deficiency can be treated.

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The right approach for SCID treatment would be to give the patient a funetioning ADA which breaks down toxic biological products.
In some children ADA deficiency could be cured by bone marroW transplantation, wher. defective immune cells could be replaced with healthy immune cells from a donor. In some patients it can be treated by enzyme replacement therapy, in which functional ADA is injected into the patient.
During gene therapy the lymphocytes from the blood of the patient are removed and grown in a utrient culture medium. A healthy and functional human gene, ADA cDNA encoding this enzyme is introduced into the lymphocytes using a retrovirus. The genetically engineered lymphocytes are subsequently returned to the patient. Since these cells are not immortal, the patient requires periodic infusion of such genetically engineered lymphocytes. The disease could be cured permanently if the gene for ADA isolated from bone marrow cells are introduced into the cells of the carly embryonic stages.
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Adenosine deaminase deficiency can be permanetly cured by which of the following methods ?

How gene therapy is done to treat ADA deficiency?

Which of the following statements is incorrect about gene therapy in ADA deficiency ?

What is gene therapy ? IIIustrate using the example of adenosine deaminase (ADA) deficiency.

Statement 1: ADA deficiency was the first disease treated by gene therapy. Statement 2: ADA is an autosomal recessive metabolie disorder.

Identify the incorrect statement. (i) The first clinical gene therapy was given by French Anderson. (ii) For a four year old boy with ADA deficiency. (iii) ADA is a autosomal dominant metabolic disorder. (iv) Where patients have non-functioning B - lymphocytes.

Select the suitable answer with respect to gene therapy from the statements given below: Statement A: ADA deficiency or SCID (Severe Combined Immunodeficiency) is an autosomal dominant metabolic disorder. Statement B : It can be cured permanently if ADA gene is introduced into the cells of early embryonic stages.