Why is tissue-specific targeting an important consideration when designing gene therapy vectors?
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Correct answer: To maximize therapeutic expression in target cells, sparing others
Tissue-specific targeting delivers the therapeutic gene mainly to diseased cells, raising efficacy and reducing off-target toxicity in other tissues. Vector tropism and promoter choice are key to achieving this specificity.
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More Gene therapy basics questions
- Which of the following is a non-viral method of gene delivery in gene therapy?
- What is the difference between gene therapy and traditional drug therapy?
- Which of the following diseases would be most amenable to gene therapy using ex vivo modification of hematopoietic stem cells?
- What is one ethical concern unique to germline gene therapy (modifying sperm, eggs, or embryos) as opposed to somatic cell gene therapy?
- Which regulatory challenge is commonly associated with clinical application of gene therapy?
- Which of the following best describes why a “perfect vector” for gene therapy does not yet exist?