The immune disorder adenosine deaminase (ADA)deficiency is the result of a mutation in one gene.How could gene therapy be used to treat this disorder?
A) Red blood cells could be induced to produce the protein encoded by the mutated ADA gene.
B) Cells containing a corrected ADA gene could be injected into patients with ADA.
C) The patients could swallow synthetic blood cells.
D) Patients could be injected with a hybrid molecule made of the DNA and RNA of the normal ADA gene.

Question

Quizplus · Accepted Answer

Gene therapy for ADA deficiency involves introducing a corrected ADA gene into the patient's cells. This can be done by extracting immune cells from the patient, correcting the ADA gene in vitro, and then re-infusing the corrected cells back into the patient. Alternatively, viral vectors carrying the corrected ADA gene can be directly injected into the patient's body. Red blood cells do not have the necessary machinery to produce the ADA protein, synthetic blood cells do not exist, and injecting a hybrid DNA/RNA molecule is not a practical gene therapy approach.

The Immune Disorder Adenosine Deaminase (ADA)deficiency Is the Result of a Mutation