Preferred Label : Autologous Base Edited CD34 HSPCs BEAM-101;
NCIt synonyms : Autologous CD34 Base Edited Hematopoietic Stem Cells BEAM-101;
NCIt definition : A population of autologous cluster of differentiation 34 (CD34)-positive human hematopoietic
stem and progenitor cells (HSPCs) ex-vivo base-edited with fusions of a deaminase
and clustered regularly interspaced short palindromic repeats (CRISPR)-Cas ribonucleoprotein,
with potential usage for transplantation in patients with sickle cell disease (SCD).
CD34-positive HSPCs are isolated from human blood upon apheresis and are genetically
modified and base-edited ex vivo to disrupt the HBG1/HBG2 gene promoter motif bound
by the transcriptional repressor B-cell lymphoma/leukemia 11A (BCL11A) gene. This
increases the expression of fetal hemoglobin (HbF) and inhibits hemoglobin S (HbS)
polymerization in erythrocytes that differentiate from BEAM-101. Upon infusion back
into the patient following myeloablative, conditioning chemotherapy, BEAM-101 can
populate the bone marrow and differentiate into a variety of blood cell types including
lymphoid cells, myeloid cells and erythroblasts. The increased production of high
levels of HbF in red blood cells (RBCs) compensates for the defective or reduced 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.;
Molecule name : BEAM 101; BEAM-101;
NCI Metathesaurus CUI : CL1906829;
Origin ID : C199468;
UMLS CUI : C5854422;
Semantic type(s)
- Cell [UMLS semantic type]
- concept_is_in_subset
