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The γ (gamma)-globin chain is part of the fetal hemoglobin, and γ-globin expression decreases after birth due to repression caused by the BCL11A transcriptional repressor. Researchers discovered that reactivation of the fetal hemoglobin production can reduce sickling of red blood cells caused by the mutant β (beta)-globin gene that encodes the adult hemoglobin chain. Which of the following gene therapies would be effective for treating sickle-cell disease?

  1. CRISPR using sgRNA specific for BCL11A to cause a knockout
  2. CRISPR using sgRNA specific for γ-globin to cause a knockout
  3. Delivery of the wild-type BCL11A transgene to red blood cells
  4. RNAi to knock down the expression of γ-globin
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The γ (gamma)-globin chain is part of the fetal hemoglobin, and γ-globin expression decreases after birth due to repression caused by the BCL11A transcriptional repressor. Researchers discovered that reactivation of the fetal hemoglobin production can reduce sickling of red blood cells caused by the mutant β (beta)-globin gene that encodes the adult hemoglobin chain. Which of the following gene therapies would be effective for treating sickle-cell disease?
TopicAll topics
SubjectBiology
ClassClass 11