Preferred Label : Autologous TNS9.3.55-transduced CD34-positive Cells;
NCIt synonyms : Autologous CD34-positive Cells Transduced with TNS9.3.55;
NCIt definition : A preparation of autologous, CD34-positive hematopoietic progenitor cells (HPCs) ex
vivo transduced with TNS9.3.55, a lentiviral vector encoding the human beta-globin
(hemoglobin-beta, HBB) gene, with potential to restore beta-globin expression and
function. Autologous CD34-positive stem cells are isolated from the patient's own
bone marrow, the deficient HBB gene is removed, and the cells are transduced with
the lentiviral vector. Upon re-infusion of the TNS9.3.55-transduced CD34-positive
cells back into the patient, these cells express beta-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.;
NCI Metathesaurus CUI : CL446651;
Origin ID : C116734;
UMLS CUI : C3899992;
Semantic type(s)
- Cell [UMLS semantic type]
- chemical_or_drug_affects_cell_type_or_tissue
- chemical_or_drug_has_mechanism_of_action
- chemical_or_drug_has_physiologic_effect
- concept_is_in_subset
