What is gene therapy? Illustrate using the example of adenosine deaminase (ADA) deficiency.
What is gene therapy? Illustrate using the example of adenosine deaminase (ADA) deficiency.
Gene therapy is a replacement of a defective or faulty gene with a normal or healthy gene, to correct a genetic disorder.
Adenosine deaminase (ADA) deficiency is caused due to the deletion of the ADA gene, coding this enzyme.
To correct this disorder:
(i) Lymphocytes are isolated from the blood or bone marrow of the patient and grown in vitro culture.
(ii) The faulty ADA gene is removed and in situ hybridization of normal ADA gene is done through transformation techniques using Adenoviral vectors.
(iii) The transformed lymphocytes are subsequently transferred to the patient under aseptic conditions.
(iii) Since, lymphocytes have a limited life span of 25-30 days, the patient requires a repeated infusion of such genetically engineered lymphocytes.
(iv) However, if the gene isolated from bone marrow cells producing ADA is introduced at early embryonic stages, it could be a permanent cure which is unethical.
Gene therapy is a replacement of a defective or faulty gene with a normal or healthy gene, to correct a genetic disorder.
Adenosine deaminase (ADA) deficiency is caused due to the deletion of the ADA gene, coding this enzyme.
To correct this disorder:
(i) Lymphocytes are isolated from the blood or bone marrow of the patient and grown in vitro culture.
(ii) The faulty ADA gene is removed and in situ hybridization of normal ADA gene is done through transformation techniques using Adenoviral vectors.
(iii) The transformed lymphocytes are subsequently transferred to the patient under aseptic conditions.
(iii) Since, lymphocytes have a limited life span of 25-30 days, the patient requires a repeated infusion of such genetically engineered lymphocytes.
(iv) However, if the gene isolated from bone marrow cells producing ADA is introduced at early embryonic stages, it could be a permanent cure which is unethical.
