Gene therapy basics · USMLE Step 1

Which regulatory challenge is commonly associated with clinical application of gene therapy?

  1. Standard dosing of small molecules
  2. Need for long-term safety monitoring over years
  3. Lack of any immune response risk
  4. Ease of reversing the therapy if side-effects occur
Show answer and explanation

Correct answer: Need for long-term safety monitoring over years

Because gene therapy can permanently change cells, long-term follow-up is needed to detect delayed adverse effects such as insertional mutagenesis or immunogenicity, making post-treatment surveillance critical.

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