What is the purpose of disabling the BCL11A gene in exa-cel therapy?
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 potential long-term complication associated with gene-altering therapies for sickle-cell disease?
A. Increased risk of vaso-occlusive crises
B. Development of blood cancers
C. Ineffective production of fetal haemoglobin
D. Loss of natural DNA-repair mechanisms
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