Preferred Label : Autologous CD34-positive BCL11A-disrupted Hematopoietic Progenitor Cells ST-400;
NCIt synonyms : Autologous CD34-positive BCL11A-disrupted HPCs ST-400; Autologous BCL11A-disrupted Hematopoietic Progenitor Cells ST-400; Autologous BCL11A-disrupted HPCs ST-400;
NCIt definition : A population of autologous CD34-positive, B-cell lymphoma/leukemia 11A (BCL11A)-disrupted
hematopoietic progenitor cells (HPCs) that may be used for bone marrow autotransplantation.
CD34-positive HPCs are isolated from human blood upon apheresis and are genetically
modified in vitro using a proprietary zinc finger nuclease (ZFN) technology to specifically
disrupt the transcriptional enhancer sequence for the BCL11A gene. Upon infusion into
the patient following conditioning chemotherapy, the autologous CD34-positive BCL11A-disrupted
HPCs ST-400 can populate the bone marrow and differentiate into a variety of blood
cell types including lymphoid cells, myeloid cells and erythroblasts. BCL11A is a
suppressor of fetal hemoglobin (HbF) expression. Therefore, disruption of the BCL11A
enhancer decreases the expression of BCL11A and stimulates the expression of HbF in
erythrocytes that differentiate from ST-400. HbF may compensate for reduced or absent
expression of adult hemoglobin in patients, including those with transfusion-dependent
beta-thalassemia.;
Molecule name : ST 400; ST-400;
NCI Metathesaurus CUI : CL550831;
Origin ID : C148240;
UMLS CUI : C4724925;
Semantic type(s)
- Cell [UMLS semantic type]
- concept_is_in_subset
