5 Questions
What is the main protein that causes red blood cells to become hard and sticky in patients with sickle cell disease?
Abnormal adult haemoglobin (HbA)
What is the gene that is edited using CRISPR/Cas9 in the gene therapy for sickle cell disease?
BCL11A
What is the name of the cell-based gene therapy that uses CRISPR/Cas9 to modify blood stem cells of patients with sickle cell disease?
Casgevy
Where are the blood stem cells transplanted back into the patient after being modified with CRISPR/Cas9?
Bone marrow
What is the main protein produced by the modified blood stem cells that assists in oxygen delivery and prevents the sickling of red blood cells?
HbF (fetal haemoglobin)
Learn about the first gene therapies approved to treat sickle cell disease, how patients with the condition have abnormal adult haemoglobin proteins, and how stimulating red blood stem cells to produce fetal haemoglobin can be an effective treatment option. Explore Casgevy, a cell-based gene therapy utilizing CRISPR/Cas9 technology.
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