Gene therapy basics · USMLE Step 1

Which strategy describes “ex vivo” gene therapy?

  1. Injecting naked plasmid DNA straight into the bloodstream
  2. Delivering a viral vector to target tissue inside the patient
  3. Modifying the patient's own cells in culture, then re-infusing them
  4. Administering small interfering RNA (siRNA) by mouth
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Correct answer: Modifying the patient's own cells in culture, then re-infusing them

Ex vivo gene therapy removes the patient's cells (e.g. stem or immune cells), modifies them in culture by gene addition or editing, and re-infuses them. It is used in some blood disorders and cancers.

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