Gene therapy basics · USMLE Step 1

How can gene therapy address diseases caused by a dominant negative mutation (where a mutant gene product interferes with normal function)?

By adding another copy of the normal gene without touching the mutant
By silencing or knocking down the mutant allele's expression
By ignoring the mutant allele, since such mutations self-correct
By delivering the gene exclusively through non-viral vectors

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Correct answer: By silencing or knocking down the mutant allele's expression

In dominant-negative disorders, silencing the mutant allele (e.g. via RNA interference or gene disruption) suppresses the harmful product's interference with normal function — an established gene therapy approach.

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How can gene therapy address diseases caused by a dominant negative mutation (where a mutant gene product interferes with normal function)?

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