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Paragraph 2 - Gene Therapy Design and Mechanism of Action layout
what casgevy doesn’t do
what its designed to do
what it can replace
whatapproach does it use
what kind of cells do they use
what happens to the cells
Paragraph 2
Gene Therapy Design and Mechanism of Action
Paragraph 2 - Gene Therapy Design and Mechanism of Action layout 1
what casgevy doesn’t do
Paragraph 2 - Gene Therapy Design and Mechanism of Action layout 2
what its designed to do
Paragraph 2 - Gene Therapy Design and Mechanism of Action layout 3
what it can replace
Paragraph 2 - Gene Therapy Design and Mechanism of Action layout 4
whatapproach does it use
Paragraph 2 - Gene Therapy Design and Mechanism of Action layout 5
what kind of cells do they use
Paragraph 2 - Gene Therapy Design and Mechanism of Action layout 6
what happens to the cells
Paragraph 2 - Gene Therapy Design and Mechanism of Action what casgevy doesn’t do
directly repair the mutant HBB gene
Paragraph 2 - Gene Therapy Design and Mechanism of Action what its designed to do
circumvent the defect by reactivating fetal haemoglobin (HbF),
Paragraph 2 - Gene Therapy Design and Mechanism of Action what it can replace
the function of the mutant adult haemoglobin.
Paragraph 2 - Gene Therapy Design and Mechanism of Action whatapproach does it use
ex vivo
Paragraph 2 - Gene Therapy Design and Mechanism of Action what kind of cells do they use
patients CD34+ haematopoietic stem cells
Paragraph 2 - Gene Therapy Design and Mechanism of Action what happens to the cells
collected and edited in a laborator