CRISPR and Sickle-Cell Disease Quiz
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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
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
What is the main function of fetal haemoglobin in the treatment of sickle-cell disease?
A. To alleviate pain
B. To prevent clumping of blood cells
C. To enable oxygen transport
D. To repair DNA sequences
What is the role of the Cas9 enzyme in the exa-cel therapy?
A. To cut both strands of DNA
B. To guide the enzyme to the target stretch of DNA
C. To stitch the DNA strands back together
D. To disable the BCL11A gene
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
What is the potential risk 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
What is the potential risk associated with modifying blood stem cells in gene-altering therapies?
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
How long are trial participants of exa-cel proposed to be followed for?
A. 5 years
B. 10 years
C. 15 years
D. 20 years
What is the primary cause of sickle-cell disease?
A. Misshapen and sticky blood cells
B. Clogged blood vessels
C. Lack of fetal haemoglobin
D. Abnormal forms of haemoglobin
What is the purpose of the CRISPR-based therapy exa-cel?
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
What is the potential risk associated with 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
What is the potential risk associated with 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
How many participants were involved in the clinical trial of exa-cel?
A. 9
B. 19
C. 29
D. 39
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
What is the main function of the CRISPR–Cas9 genome editing system in exa-cel therapy?
A. To cut both strands of DNA
B. To guide the enzyme to the target stretch of DNA
C. To stitch the DNA strands back together
D. To disable the BCL11A gene
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