explain enzyme replacement therapy to treat ADA deficiency.mention 2 disadvantages of this procedure.

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Please find below the solution to the asked query

Adenosine deaminase (ADA) is an enzyme crucial for immune system. Its deficiency is caused due to the deletion of the gene coding for adenosine deaminase.

Steps involved in gene therapy for ADA deficiency are:

  1. Lymphocytes isolated from patient’s blood are cultured in-vitro.
  2. Functional ADA cDNA are then introduced into the cultured lymphocytes.
  3. These lymphocytes are returned back to the patient’s body.
Lymphocytes are not immortal. Therefore, repeated infusion of genetically engineered lymphocytes is required. However, if the gene isolated from bone marrow cells is introduced at embryonic stage, the disease can be cured permanently.


  1. It is very costly
  2. The patient requires repeated infusion of genetically engineered lymphocytes.

Hope this information will clear your doubts about the ADA gene therapy.
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