Zynteglo

Zynteglo - a preparation of autologous, cd34-positive hematopoietic stem cells (hscs) transduced ex vivo with the bb305 recombinant replication-defective, self-inactivating lentiviral vector encoding for an engineered form of human beta-globin (hemoglobin-beta, hbb) gene, beta-a-t87q (b-a-t87q) where the threonine at position 87 has been substituted with glutamine, with potential to restore beta-globin expression and function. Autologous cd34-positive stem cells are isolated from the patient's own bone marrow and the cells are transduced with the lentiviral vector. Upon re-infusion of betibeglogene autotemcel back into the patient, these cells express b-a-t87q-globin, thereby allowing the body to make normal hemoglobin and thus normal, healthy red blood cells. Beta-globin, the beta-chain of the most common form of hemoglobin, is encoded by the hbb gene; mutations in this gene prevent normal beta-globin production and are associated with beta-thalassemia and sickle cell anemia. The b-a-t87q form of beta-globin has increased antisickling activity compared to the wild type protein. See betibeglogene autotemcel

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Source: Data courtesy of the U.S. National Library of Medicine. Since the data might have changed, please query MeSH on Zynteglo for any updates.

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