Gene therapy basics · USMLE Step 1

Which of the following best describes why a “perfect vector” for gene therapy does not yet exist?

  1. Because no known virus is able to enter human cells
  2. Because every vector trades off efficiency, capacity, targeting, and safety
  3. Because non-viral methods always integrate genes into host DNA
  4. Because current vectors work only in laboratory animals
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Correct answer: Because every vector trades off efficiency, capacity, targeting, and safety

Every delivery vector has trade-offs — viral vectors may integrate unpredictably or provoke immune responses, while non-viral methods may be inefficient or poorly targeted — so no single vector is universally optimal.

Difficulty: Medium Question 16 of 21

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