Question

What is the first step in gene therapy to treat ADA deficiency?

• A. Blood transfusions using genetically engineered lymphocytes.
• B. Artificial culturing of the lymphocytes from the patient.
• C. Production of genetically engineered lymphocytes.
• D. Introduction of a cDNA coding for functional ADA gene into the lymphocytes.

Answer 1

The correct option is B

Artificial culturing of the lymphocytes from the patient.

The first step in gene therapy to treat ADA deficiency is to artificially grow the lymphocytes from the blood of the patients in culture. Adenosine deaminase is required for the lymphocytes to mature. As gene therapy aims to replace the corrupted copy of the gene, it needs to be done in the cells where it is targeted. Therefore, culturing of lymphocytes is the first step in the treatment of this disease by gene therapy.