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Gene therapy basics · USMLE Step 1

What is the difference between gene therapy and traditional drug therapy?

  1. Gene therapy alters the patient's genes; drugs act via external molecules
  2. Gene therapy is always reversible, unlike conventional drug therapy
  3. Drug therapy edits DNA directly, whereas gene therapy uses proteins
  4. The two are essentially identical in their mechanism of action
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Correct answer: Gene therapy alters the patient's genes; drugs act via external molecules

Gene therapy works at the genetic level — adding or altering genes in the patient's cells — whereas traditional drug therapy uses chemicals that modulate cellular processes without changing the genome.

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