A person is born with a hereditary disease with a weakened immune system due to deficiency of an enzyme. Suggest a technique for complete cure for this disease, identify the deficient enzyme and explain the technique used for cure.
If a person is born with a hereditary disease with a weakened immune system, he/she can be treated by a technique known as gene therapy. The detection of the deficiency is done at an early stage only that is in a child or embryo. As it is a genetic defect, diagnosis and treatment are done at the genetic level. The deficient genes in normal condition are inserted in the patient's cells or tissues. This helps to overcome the non-functionality of the defective genes. The hereditary disease referred to in the question is due to the deficiency of ADA or adenosine deaminase enzyme. This enzyme contributes to the proper functioning of the immune system. ADA deficiency was first reported in 1990 in a 4-year-old girl.
ADA deficiency can be cured by bone marrow transplantation or enzyme replacement therapy. The functional ADA enzyme is injected into the patient. But these two approaches do not provide a complete cure. Hence gene therapy is used. The white blood corpuscles or lymphocytes are taken from the patient’s blood and cultured outside in the laboratory. Then ADA cDNA(complementary DNA) is introduced in the white blood corpuscles and then is transferred or injected into the patient. The patient, however, requires periodic transfusion of this genetically engineered white blood corpuscles.
Couldn't generate an explanation.
Generated by AI. May contain inaccuracies — always verify with your textbook.
