can a corrective therapy be takenfor a hereditary disease

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Please find below the solution to the asked query

Gene therapy is a possible corrective therapy that can be taken to correct a genetic or congenital diseases. The topic of gene therapy is still new and emerging medical technology. Till now many clinical trails of these therapies have been conducted or approved. It involves the delivery of a normal gene into the individual to replace the defective gene.  The first disease that was treated by gene therapy was ADA (Adenosine deaminase) deficiency. In this, the gene coding for enzyme ADA becomes non functional that causes ADA. The ADA is important for normal functioning of the immune system

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.

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