Q. With reference to the CRISPR-Cas9 Gene Therapy in the context of sickle cell disease, which of the following statements are correct?
1.The primary genetic defect that causes sickle cell disease is the mutation in the hemoglobin B gene.
2.CRISPR-Cas9 gene editing aims to treat sickle cell disease by introducing beneficial mutations in the HBB gene to mimic hereditary persistence of fetal hemoglobin (HPFH).
3.The function of the Cas9 protein in the CRISPR-Cas9 system is that it acts as a guide RNA to locate the target DNA sequence.
Select the correct answer using the codes given below:
Explanation –
Statements 1 and 2 are correct. Sickle cell disease is caused by a mutation in the HBB gene that instructs the body to produce abnormal hemoglobin. CRISPR-Cas9 can be used to introduce various modifications to address sickle cell disease, including potentially correcting the mutation or introducing changes to promote fetal hemoglobin production. India has aimed to eradicate sickle cell disease by 2047.
Statement 3 is incorrect. In the CRISPR-Cas9 system, the Cas9 protein acts as a nuclease (molecular scissor) that cleaves the target DNA at a specific location guided by the guide RNA. The guide RNA molecule itself recognizes and binds to the target DNA sequence.
Source: The Hindu
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