Preferred Label : Autologous gamma-globinG16D/sh RNA734 Gene-transduced CD34-positive Cells CSL200;
NCIt synonyms : Human gamma-globinG16D/Short-hairpin RNA734-transduced CD34-positive Cells CSL200;
NCIt definition : A preparation of autologous, CD34-positive hematopoietic stem cells (HSCs) transduced
ex vivo with a lentiviral vector encoding for an engineered form of human gamma-globin
(hemoglobin-gamma) gene, gamma-globin G16D, and a short-hairpin (sh) RNA734, with
potential to restore gamma-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 CD34-positive cells back into
the patient, these cells express gamma-globinG16D, thereby replacing defective beta
globin chains with gamma globin chains allowing the body to make fetal hemoglobin
(HbF) and thus healthy red blood cells with a greater oxygen-carrying capacity. Gamma-globin
comprises the gamma-chain of HbF; reactivation of HbF in disorders where beta-globin
is defective in adult hemoglobin (HbA), such as sickle cell disease and beta thalassemia,
may ameliorate these conditions. The G16D form of gamma-globin has increased anti-sickling
activity compared to the wild type protein. The shRNA734 is used for positive gene
selection.;
Molecule name : CSL 200; CSL-200;
NCI Metathesaurus CUI : CL978619;
Origin ID : C165434;
UMLS CUI : C5237177;
Semantic type(s)
- Cell [UMLS semantic type]
concept_is_in_subset