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- NCIT_C169042 IAO_0000115 "A population of autologous cluster of differentiation 34 (CD34)-positive human hematopoietic stem and progenitor cells (hHSPCs) that are ex-vivo gene-edited with the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nuclease complex at the erythroid lineage-specific enhancer of the B-cell lymphoma/leukemia 11A (BCL11A) gene, with potential usage for transplantation in patients with sickle cell disease (SCD) and beta-thalassemia. CD34-positive HSPCs are isolated from human blood upon apheresis and are genetically modified in vitro using CRISPR/Cas9 technology to specifically disrupt the erythroid enhancer of the BCL11A gene. As BCL11A is a suppressor of fetal hemoglobin (HbF; hemoglobin F) expression, disruption of the BCL11A enhancer decreases the expression of BCL11A and stimulates the expression of HbF in erythrocytes that differentiate from CTX001. Upon infusion back into the patient following myeloablative, conditioning chemotherapy, exagamglogene autotemcel 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 and beta-thalassemia. 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." @default.
- NCIT_C169042 NCIT_NHC0 "C169042" @default.
- NCIT_C169042 NCIT_P106 "Cell" @default.
- NCIT_C169042 NCIT_P106 "Pharmacologic Substance" @default.
- NCIT_C169042 NCIT_P107 "Exagamglogene Autotemcel" @default.
- NCIT_C169042 NCIT_P108 "Exagamglogene Autotemcel" @default.
- NCIT_C169042 NCIT_P208 "CL1378853" @default.
- NCIT_C169042 NCIT_P319 "S53L777GM8" @default.
- NCIT_C169042 NCIT_P322 "CTRP" @default.
- NCIT_C169042 NCIT_P322 "FDA" @default.
- NCIT_C169042 NCIT_P329 "801170" @default.
- NCIT_C169042 NCIT_P330 "801170" @default.
- NCIT_C169042 NCIT_P399 "801170" @default.
- NCIT_C169042 normalizedInformationContent "100" @default.
- NCIT_C169042 referenceCount "1" @default.
- NCIT_C169042 hasExactSynonym "Autologous CD34+ HSPCs Modified with CRISPR-Cas9 at the Erythroid Lineage-specific Enhancer of the BCL11A Gene" @default.
- NCIT_C169042 hasExactSynonym "Autologous CD34-positive BCL11A Enhancer-disrupted HSPCs CTX001" @default.
- NCIT_C169042 hasExactSynonym "Autologous CRISPR-Cas9 Engineered CD34+ hHSPCs CTX001" @default.
- NCIT_C169042 hasExactSynonym "Autologous CRISPR-Cas9 Genetically-modified CD34+ HSPCs CTX001" @default.
- NCIT_C169042 hasExactSynonym "Autologous CRISPR-Cas9 Modified/ BCL11A Enhancer-disrupted CD34-positive HSPCs CTX001" @default.
- NCIT_C169042 hasExactSynonym "Autologous CRISPR-Cas9 Modified/BCL11A Enhancer-disrupted CD34-positive Human Hematopoietic Stem and Progenitor Cells CTX001" @default.
- NCIT_C169042 hasExactSynonym "CTX 001" @default.
- NCIT_C169042 hasExactSynonym "CTX-001" @default.
- NCIT_C169042 hasExactSynonym "CTX001" @default.
- NCIT_C169042 hasExactSynonym "EXAGAMGLOGENE AUTOTEMCEL" @default.
- NCIT_C169042 hasExactSynonym "Exagamglogene Autotemcel" @default.
- NCIT_C169042 inSubset NCIT_C116977 @default.
- NCIT_C169042 inSubset NCIT_C116978 @default.
- NCIT_C169042 inSubset NCIT_C176424 @default.
- NCIT_C169042 inSubset NCIT_C63923 @default.
- NCIT_C169042 type Class @default.
- NCIT_C169042 isDefinedBy ncit.owl @default.
- NCIT_C169042 label "Exagamglogene Autotemcel" @default.
- NCIT_C169042 subClassOf NCIT_C142078 @default.
- NCIT_C169042 subClassOf NCIT_C169042 @default.
- NCIT_C169042 subClassOf NCIT_C1908 @default.
- NCIT_C169042 subClassOf NCIT_C1909 @default.
- NCIT_C169042 subClassOf NCIT_C201554 @default.
- NCIT_C169042 subClassOf NCIT_C307 @default.
- NCIT_C169042 subClassOf NCIT_C308 @default.