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.