A research team is developing a CRISPR-Cas9
therapy for sickle cell disease using a patient’s haematopoietic stem
cells. Instead of trying to directly fix the mutated β-globin gene, they design
a strategy that edits a regulatory region
controlling haemoglobin expression . After editing, patients begin
producing high levels of fetal haemoglobin
(HbF) , which compensates for the defective adult β-globin.

Which gene-editing strategy best explains how
this therapy works?

Question

A research team is developing a  CRISPR-Cas9
therapy for sickle cell disease   using a patient’s haematopoietic stem
cells. Instead of trying to directly fix the mutated β-globin gene, they design
a strategy that edits a  regulatory region
controlling haemoglobin expression . After editing, patients begin
producing high levels of  fetal haemoglobin
(HbF) , which compensates for the defective adult β-globin.

Which gene-editing strategy best explains how
this therapy works?

Accepted Answer

Disrupting
the BCL11A erythroid enhancer in haematopoietic stem cells using non-homologous
end joining (NHEJ), allowing fetal haemoglobin expression

Answer

Inserting
an additional functional β-globin gene into haematopoietic stem cells using
CRISPR-mediated recombination

Answer

Repairing
the regulatory enhancer controlling BCL11A using HDR to restore normal β-globin
expression

Answer

Precisely
correcting the mutated β-globin gene sequence using homology-directed repair
(HDR)

Crowdly

A research team is developing a CRISPR-Cas9 therapy for sickle cell disease ...

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