What is the purpose of the exa-cel therapy in sickle-cell disease?
A. To cure sickle-cell disease
B. To alleviate pain caused by sickle-cell disease
C. To increase the production of fetal haemoglobin
D. To prevent vaso-occlusive crises
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What is the main concern regarding off-target edits in CRISPR–Cas9 therapies?
A. Introduction of unwanted mutations
B. Increased risk of cancer
C. Inadequate genetic diversity in the analysis
D. Insufficient sampling of cells from sickle-cell disease patients
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