Gene therapy basics · USMLE Step 1

Which of the following is an advantage of using in vivo gene therapy over ex vivo gene therapy?

  1. It completely avoids any immune response in all cases
  2. It can reach hard-to-harvest tissues such as neurons
  3. It still requires cell culture and later reinfusion
  4. It is fully and easily reversible after treatment
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Correct answer: It can reach hard-to-harvest tissues such as neurons

In vivo gene therapy delivers genetic material directly to target tissues in the body, which is advantageous for cells that are hard to harvest (e.g. neurons, muscle) and avoids cell removal and reinfusion.

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