Question

The genetic defect-adenosine deaminase (ADA) deficiency may be cured permanently by

• A. Enzyme replacement therapy
• B. Periodic infusion of genetically engineered lymphocytes having functional ADA cDNA
• C. Administering adenosine deaminase activators
• D. Introducing bone marrow cells producing ADA into cells at early embryonic stages

Answer 1

The correct option is D Introducing bone marrow cells producing ADA into cells at early embryonic stages

Gene therapy is the process of introduction of DNA into living human beings in order to treat disease. It is used to replace a missing gene product or to correct mutant alleles permanently. The ex vivo introduction of functional ADA gene in bone marrow cells of the patient, suffering from SCID, through an engineered retrovirus containing a functional ADA gene followed by reintroduction of treated cells into the patient’s marrow treat the disease permanently.