Casgevy preparation procedures are as follows:19
- Step 1: Obtain blood stem cells that express the stem cell biomarker called CD34 from the sickle cell anemia patient to silence BCL11A by CRISPR/Cas9.
- Step 2: The silencing of BCL11A releases fetal hemoglobin (Hb F) from suppression and begins to produce fetal hemoglobin proteins, which are more efficient oxygen binders than adult hemoglobin (Hb A) and can, hence, more efficiently transport and distribute oxygen to the rest of the body as blood circulates.
- Step 3: The CRISPR/Cas9-edited stem cells are then transplanted back to the patient, where the edited cells adhere and proliferate in the bone marrow, leading to sizable quantities of fetal hemoglobin. This procedure helps edited cells outcompete sickle-shaped nonfunctional RBCs to restore normal oxygen-binding, carrying, and distribution. A team of medical professionals closely monitors patients who have received Casgevy therapy.
19. Davies, K., PhD. (2024, February 1). FDA approves the first CRISPR therapy for sickle cell disease. GEN - Genetic Engineering and Biotechnology News. https://www.genengnews.com/topics/genome-editing/fda-approves-the-first-crispr-therapy-for-sickle-cell-disease/
