Gene therapy basics · USMLE Step 1

Which of the following describes an “in vivo” gene therapy approach?

  1. Gene is modified in cultured cells, then transplanted back into the patient
  2. Genetic material is delivered directly into the patient’s body
  3. Only animal studies are done
  4. Gene therapy applied to bacterial infections
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Correct answer: Genetic material is delivered directly into the patient’s body

In vivo gene therapy delivers the therapeutic genetic material directly into the patient's body to transduce target cells in place, unlike ex vivo approaches that modify cells outside the body first.

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