ADA-Adenosine Deaminase. It is required for the proper functioning of immune system. Gene therapy for ADA deficiency are: Gene therapy is helpful in case of ADA deficiency. 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.
Expand the name of the enzyme ADA. Why is the enzyme essential in the human body? Suggest a gene therapy for its deficiency.
Answers
Anki Jain
ADA-Adenosine Deaminase. It is required for the proper functioning of immune system.
Gene therapy for ADA deficiency are:
Gene therapy is helpful in case of ADA deficiency.
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.