Hereditary disease can be corrected by gene therapy. It is a collection of methods that allows correction or replacement of defective gene. The first gene therapy was given in 1990 to a 4 years old girl with Adenosine Deaminase (ADA) deficiency. It is caused due to the deletion of gene for adenosine deaminase.
The treatment involves following steps: (i) Lymphocytes from the blood of patient are grown on culture outside the body. (ii) A functional ADA, cDNA (using a Retro viral vector) is then introduced into these lymphocytes. (iii) Such genetically engineered lymphocytes are returned to the blood of patient. (iv) Periodic infusion of such genetically engineered lymphocyte is required by the patient.
How is gene therapy being used in treating ADA deficiency patients?
Answers
ABHISHEK GUPTA
Hereditary disease can be corrected by gene therapy. It is a collection of methods that allows correction or replacement of defective gene. The first gene therapy was given in 1990 to a 4 years old girl with Adenosine Deaminase (ADA) deficiency. It is caused due to the deletion of gene for adenosine deaminase.
The treatment involves following steps:
(i) Lymphocytes from the blood of patient are grown on culture outside the body.
(ii) A functional ADA, cDNA (using a Retro viral vector) is then introduced into these lymphocytes.
(iii) Such genetically engineered lymphocytes are returned to the blood of patient.
(iv) Periodic infusion of such genetically engineered lymphocyte is required by the patient.