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- NCIT_C157393 IAO_0000115 "A population of autologous cluster of differentiation 34 (CD34)-positive hematopoietic progenitor cells (HPCs) that are transfected ex vivo with zinc finger nuclease (ZFN) messenger ribonucleic acid (mRNA) targeting the B-cell lymphoma/leukemia 11A (BCL11A) locus, with potential usage for transplantation in patients with sickle cell disease (SCD). CD34-positive HPCs are isolated from human blood upon apheresis and are genetically modified in vitro using ZFN technology to specifically cleave and disrupt the erythroid enhancer of the BCL11A gene. This suppresses the production of sickle hemoglobin. Upon infusion into the patient following conditioning chemotherapy, the autologous CD34-positive BCL11A-disrupted HPCs BIVV003 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) expression, disruption of the BCL11A enhancer decreases the expression of BCL11A and stimulates the expression of HbF in erythrocytes that differentiate from BIVV003. HbF may compensate for reduced or absent expression of adult hemoglobin in patients with SCD." @default.
- NCIT_C157393 NCIT_NHC0 "C157393" @default.
- NCIT_C157393 NCIT_P106 "Cell" @default.
- NCIT_C157393 NCIT_P106 "Pharmacologic Substance" @default.
- NCIT_C157393 NCIT_P107 "Autologous CD34-positive BCL11A-disrupted Hematopoietic Progenitor Cells BIVV003" @default.
- NCIT_C157393 NCIT_P108 "Autologous CD34-positive BCL11A-disrupted Hematopoietic Progenitor Cells BIVV003" @default.
- NCIT_C157393 NCIT_P208 "CL936984" @default.
- NCIT_C157393 NCIT_P322 "CTRP" @default.
- NCIT_C157393 NCIT_P329 "796965" @default.
- NCIT_C157393 NCIT_P330 "796965" @default.
- NCIT_C157393 NCIT_P399 "796965" @default.
- NCIT_C157393 normalizedInformationContent "100" @default.
- NCIT_C157393 referenceCount "1" @default.
- NCIT_C157393 hasExactSynonym "Autologous CD34+ Hematopoietic Stem Cells BIVV003" @default.
- NCIT_C157393 hasExactSynonym "Autologous CD34-positive BCL11A-disrupted HPCs BIVV003" @default.
- NCIT_C157393 hasExactSynonym "Autologous CD34-positive BCL11A-disrupted Hematopoietic Progenitor Cells BIVV003" @default.
- NCIT_C157393 hasExactSynonym "BIVV003" @default.
- NCIT_C157393 hasExactSynonym "HSPCs BIVV003" @default.
- NCIT_C157393 inSubset NCIT_C116977 @default.
- NCIT_C157393 inSubset NCIT_C116978 @default.
- NCIT_C157393 inSubset NCIT_C176424 @default.
- NCIT_C157393 type Class @default.
- NCIT_C157393 isDefinedBy ncit.owl @default.
- NCIT_C157393 label "Autologous CD34-positive BCL11A-disrupted Hematopoietic Progenitor Cells BIVV003" @default.
- NCIT_C157393 subClassOf NCIT_C142078 @default.
- NCIT_C157393 subClassOf NCIT_C157393 @default.
- NCIT_C157393 subClassOf NCIT_C1908 @default.
- NCIT_C157393 subClassOf NCIT_C1909 @default.
- NCIT_C157393 subClassOf NCIT_C201554 @default.
- NCIT_C157393 subClassOf NCIT_C307 @default.
- NCIT_C157393 subClassOf NCIT_C308 @default.