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 BCL11A-disrupted human hematopoietic stem and progenitor cells HIX763

A population of autologous human hematopoietic stem and progenitor cells (HSPCs) that are genetically modified to disrupt the activity of B-cell lymphoma/leukemia 11A (BCL11A), with potential usage for transplantation in patients with sickle cell disease (SCD). Upon infusion into the patient, the autologous BCL11A-disrupted HSPCs HIX763 can populate the bone marrow and differentiate into a variety of blood cell types including lymphoid cells, myeloid cells and erythroblasts. As BCL11A is a suppressor of fetal hemoglobin (HbF; hemoglobin F) expression, disruption of the expression of BCL11A stimulates the expression of HbF in erythrocytes that differentiate from HIX763. HbF may compensate for reduced or absent expression of adult hemoglobin (Hb) in patients with SCD. HbF is a form of the oxygen carrying Hb that is naturally present at birth and is then replaced by the adult form of hemoglobin.
Check for active clinical trials using this agentView this agent in the NCI Thesaurus
Synonym:autologous BCL11A-disrupted HSPCs HIX763
autologous genetically-modified HSPCs HIX763
Code name:HIX 763
HIX-763
HIX763
Search NCI's Drug Dictionary
Please enter up to 30 characters for your search