Why might a patient with ADA-SCID be a good candidate for somatic cell gene therapy?
1)The ADA gene was one of the first human disease genes to be cloned and characterized.
2)White blood cells can easily be obtained from ADA - SCID patients and reintroduced after functional copies of the ADA gene are added
3)Even a small amount of functional ADA will restore partial immune function
A) 1
B) 2
C) 3
D) 1 and 2
E) All of these

Question

Quizplus · Accepted Answer

1) The early cloning and characterization of the ADA gene provide a clear target for gene therapy, making it easier to develop treatments. 2) The ability to easily obtain, modify, and reintroduce white blood cells into patients simplifies the process of gene therapy for ADA-SCID. 3) Since even a small increase in ADA activity can significantly impact immune function, gene therapy does not need to fully correct the gene defect to be beneficial.

Why Might a Patient with ADA-SCID Be a Good Candidate