What is a major concern associated with genome-editing gene therapy methods like CRISPR in clinical application?
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Correct answer: Off-target edits causing unintended mutations elsewhere
A major risk of CRISPR and other nucleases is off-target editing — unintended DNA breaks or mutations elsewhere in the genome — which is a critical safety concern in therapeutic gene editing.
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More Gene therapy basics questions
- 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?
- 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?