On December 8, 2023 the FDA approved Lyfgenia, a second gene…
On December 8, 2023 the FDA approved Lyfgenia, a second gene therapy for sickle cell disease. We did not discuss Lyfgenia in class and it does not involve CRISPR Cas9 gene editing. Instead, it uses gene therapy to deliver a modified version of the gene that encodes the hemoglobin protein to RBCs. This modified version of the hemoglobin gene produces hemoglobin protein that has an anti-sickling effect on red blood cells, even if these cells are also expressing the mutant hemoglobin protein. A serious side effect of Lyfgenia is leukemia, a type of blood cancer. Patients receiving Lyfgenia should have lifelong monitoring for such cancers. What vector does Lyfgenia most likely use to deliver the modified hemoglobin gene?
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