Deck 5: Gene Therapy and Emerging Molecular Therapies

Question 1

Which of the following is NOT a potential advantage of gene therapy over traditional pharmacologic therapy?

A) Gene therapy can replace a mutant gene that results in disease, thereby striking the root of the condition.
B) Gene therapy can result in continuous production of a therapeutic protein with a short t1/2 that would otherwise require frequent dosing.
C) Gene therapy in Phase 4 studies has been proven safer and more efficacious than analogous drug treatments.
D) Gene therapy can be targeted to a specific site or cell type to avoid potentially toxic systemic therapy.
E) Gene therapy can improve patient compliance dramatically, since it does not rely on daily adherence to a treatment.

Answer 1

Gene therapy in Phase 4 studies has been proven safer and more efficacious than analogous drug treatments.

Question 2

Choose the true statement regarding gene transfer mechanisms.

A) Plasmid DNA transfer has the advantage of being highly efficient.
B) Retrovirus transfer is optimal when the target cell is undergoing division.
C) The high rate of protein uptake makes liposomal transfer mechanisms less desirable.
D) Adenovirus transfer typically has effects lasting 3 years or longer.
E) Herpes simplex viruses are clinically used to transfer DNA to reproductive organs.

Answer 2

Retrovirus transfer is optimal when the target cell is undergoing division.