Skip to main content
An official website of the United States government
Government Funding Lapse
Because of a lapse in government funding, the information on this website may not be up to date, transactions submitted via the website may not be processed, and the agency may not be able to respond to inquiries until appropriations are enacted.

The NIH Clinical Center (the research hospital of NIH) is open. For more details about its operating status, please visit cc.nih.gov.

Updates regarding government operating status and resumption of normal operations can be found at opm.gov.

autologous CD34-positive BCL11A-disrupted hematopoietic progenitor cells ST-400

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.
Synonym:autologous BCL11A-disrupted hematopoietic progenitor cells ST-400
autologous BCL11A-disrupted HPCs ST-400
autologous CD34-positive BCL11A-disrupted HPCs ST-400
Code name:ST 400
ST-400
Search NCI's Drug Dictionary