NCI Drug Dictionary

The NCI Drug Dictionary contains technical definitions and synonyms for drugs/agents used to treat patients with cancer or conditions related to cancer. Each drug entry includes links to check for clinical trials listed in NCI's List of Cancer Clinical Trials.

autologous beta-A(T87Q)-globin gene-transduced CD34-positive cells
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 the b-A-T87Q-globin gene transduced CD34-positive cells 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. Check for active clinical trials using this agent. (NCI Thesaurus)

Synonym:autologous CD34+ HSCs transduced with LentiGlobin BB305
LentiGlobin BB305 lentiviral vector-transduced autologous CD34+ HSCs
LentiGlobin BB305-transduced CD34-positive cells
lentiviral b-A(T87Q)-globin-transduced CD34-positive cells
US brand name:LentiGlobin BB305 Drug Product