Gene therapy requires
A) knowledge and availability of the wild- type allele of the defective gene.
B) an ability to introduce the wild- type allele into the patient.
C) an ability to express the introduced gene at the correct level, time, and site within the patient.
D) A and B.
E) all of the above.

Question

Quizplus · Accepted Answer

Gene therapy requires all of the factors listed in order to be successful. Knowledge and availability of the wild-type allele of the defective gene is important to be able to identify the specific mutation causing the disease. An ability to introduce the wild-type allele into the patient is necessary to replace the mutated gene with a healthy copy. Finally, an ability to express the introduced gene at the correct level, time, and site within the patient is important to ensure that the therapy is targeted and effective. Therefore, all of the choices listed are necessary for successful gene therapy.

Gene Therapy Requires