
CRISPR and Sickle-Cell Disease
Authored by Ian Rodgers
Biology
12th Grade
Used 4+ times

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15 questions
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1.
MULTIPLE CHOICE QUESTION
30 sec • 1 pt
What is the purpose of the CRISPR-based therapy exa-cel?
To cure sickle-cell disease
To alleviate pain caused by sickle-cell disease
To increase the production of fetal haemoglobin
To prevent vaso-occlusive crises
2.
MULTIPLE CHOICE QUESTION
30 sec • 1 pt
What is the main concern regarding off-target edits in CRISPR–Cas9 therapies?
Introduction of unwanted mutations
Increased risk of cancer
Inadequate genetic diversity in the analysis
Insufficient sampling of cells from sickle-cell disease patients
3.
MULTIPLE CHOICE QUESTION
30 sec • 1 pt
What is the potential long-term complication associated with gene-altering therapies for sickle-cell disease?
Increased risk of vaso-occlusive crises
Development of blood cancers
Ineffective production of fetal haemoglobin
Loss of natural DNA-repair mechanisms
4.
MULTIPLE CHOICE QUESTION
30 sec • 1 pt
How many participants were involved in the clinical trial of exa-cel?
9
19
29
39
5.
MULTIPLE CHOICE QUESTION
30 sec • 1 pt
What is the role of the Cas9 enzyme in the exa-cel therapy?
To cut both strands of DNA
To guide the enzyme to the target stretch of DNA
To stitch the DNA strands back together
To disable the BCL11A gene
6.
MULTIPLE CHOICE QUESTION
30 sec • 1 pt
What is the main function of fetal haemoglobin in the treatment of sickle-cell disease?
To alleviate pain
To prevent clumping of blood cells
To enable oxygen transport
To repair DNA sequences
7.
MULTIPLE CHOICE QUESTION
30 sec • 1 pt
What is the potential risk associated with modifying blood stem cells in gene-altering therapies?
Increased risk of vaso-occlusive crises
Development of blood cancers
Ineffective production of fetal haemoglobin
Loss of natural DNA-repair mechanisms
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