Gene Therapy and ADA Deficiency

• It is completely curable by enzyme replacement therapy.
• It is caused by a mutation in the gene for adenosine deaminase. (correct)
• It is a disorder of the immune system.
• It can only be treated by bone marrow transplantation.

• Introducing the gene isolate from marrow cells into cells at early embryonic stages.
• Growing lymphocytes from the patient's blood in a culture outside the body. (correct)
• Injecting functional ADA into the patient.
• Infusing genetically engineered lymphocytes periodically.

• The enzyme replacement therapy does not address the underlying genetic cause. (correct)
• The patient requires periodic infusion of genetically engineered lymphocytes.
• The functional ADA is not effective in the patient's body.
• The patient's immune system rejects the injected ADA.