Explain enzyme-replacement therapy to treat adenosine deaminase defici

1.	Explain enzyme-replacement therapy to treat adenosine deaminase deficiency. Mention two disadvantages of this procedure.
Answer» Functional adenosine deaminase is given to the patient by injection. i. Lymphocytes from the blood of the patient are grown on culture outside the body. ii. A functional ADA, cDNA is then introduced into these lymphocytes using a retroviral vector. iii. The genetically engineered lymphocyte are returned to the blood of patient. Disadvantages: Therapy is not completely curative as cells do not remain alive and periodic infusion of lymphocytes is required.

Explain enzyme-replacement therapy to treat adenosine deaminase deficiency. Mention two disadvantages of this procedure.

Answer»

Functional adenosine deaminase is given to the patient by injection.

i. Lymphocytes from the blood of the patient are grown on culture outside the body.

ii. A functional ADA, cDNA is then introduced into these lymphocytes using a retroviral vector.

iii. The genetically engineered lymphocyte are returned to the blood of patient.

Disadvantages: Therapy is not completely curative as cells do not remain alive and periodic infusion of lymphocytes is required.