Gene therapy basics · USMLE Step 1

Which of the following is a potential benefit of gene editing (e.g., using CRISPR) over traditional gene addition therapy?

  1. It avoids all immune detection by using no delivery vector
  2. It corrects the mutation directly at its native gene locus
  3. It works only in non-human experimental cells
  4. Its effects are always fully reversible afterward
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Correct answer: It corrects the mutation directly at its native gene locus

Gene editing (e.g. CRISPR) can correct or disrupt a mutation directly at its native locus, restoring normal gene function and regulation, rather than adding a separate working copy as in traditional gene-addition therapy.

Difficulty: Medium Question 7 of 21

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