Gene therapy basics · USMLE Step 1

What is the primary goal of gene therapy?

  1. To change a person’s diet to reduce disease risk
  2. To modify a person’s genes to treat or cure a disease
  3. To transplant diseased cells into a healthy donor
  4. To use antibodies to neutralize pathogens
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Correct answer: To modify a person’s genes to treat or cure a disease

Gene therapy aims to modify a person's genes — adding, replacing, or editing genetic material — to treat or cure disease. This distinguishes it from dietary, transplant, or antibody-based approaches.

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