What is one major limitation of AAV vectors in gene therapy?
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Correct answer: Large genes exceed their small packaging capacity
AAV vectors have a small packaging capacity (~4.7 kb), so large therapeutic genes plus their regulatory elements often cannot fit. AAV is mostly non-integrating and can transduce non-dividing cells, so the other options are false.
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More Gene therapy basics questions
- Which of the following is a potential benefit of gene editing (e.g., using CRISPR) over traditional gene addition therapy?
- What is a major concern associated with genome-editing gene therapy methods like CRISPR in clinical application?
- Which of the following is NOT a major type of gene therapy approach?
- Why is tissue-specific targeting an important consideration when designing gene therapy vectors?
- 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?