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- NCIT_C176855 IAO_0000115 "A preparation of autologous, CD34-positive hematopoietic stem cells (HSCs), obtained from patients with sickle cell disease (SCD), transduced ex vivo with the BB305 recombinant replication-defective, self-inactivating lentiviral vector (LVV) encoding for an engineered, modified form of human beta-globin (hemoglobin-beta, HBB) gene, beta-A-T87Q (b-A-T87Q), in which the threonine (Thr; T) at position 87 has been substituted with glutamine (Gln; Q), with potential to restore beta-globin expression and function in SCD patients. Autologous CD34-positive stem cells are isolated from the patient's own bone marrow and the cells are transduced with the lentiviral vector. Upon re-infusion of lovotibeglogene autotemcel back into the patient, these cells express the non-sickling b-A-globin protein b-A-T87Q-globin, thereby allowing the body to make anti-sickling hemoglobin, HbA-T87Q, and thus normal, healthy red blood cells (RBCs). Beta-globin, the beta-chain of the most common form of hemoglobin, is encoded by the HBB gene; mutations in this gene prevent normal beta-globin production and are associated with SCD. The b-A-T87Q form of beta-globin has increased anti-sickling activity compared to the wild type protein." @default.
- NCIT_C176855 NCIT_NHC0 "C176855" @default.
- NCIT_C176855 NCIT_P106 "Cell" @default.
- NCIT_C176855 NCIT_P106 "Pharmacologic Substance" @default.
- NCIT_C176855 NCIT_P107 "Lovotibeglogene Autotemcel" @default.
- NCIT_C176855 NCIT_P108 "Lovotibeglogene Autotemcel" @default.
- NCIT_C176855 NCIT_P208 "CL1642730" @default.
- NCIT_C176855 NCIT_P319 "2C6A9NH2Z8" @default.
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- NCIT_C176855 NCIT_P329 "803810" @default.
- NCIT_C176855 NCIT_P330 "803810" @default.
- NCIT_C176855 NCIT_P399 "803810" @default.
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- NCIT_C176855 NCIT_R72 NCIT_C104083 @default.
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- NCIT_C176855 NCIT_R72 NCIT_C33061 @default.
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- NCIT_C176855 normalizedInformationContent "100" @default.
- NCIT_C176855 referenceCount "1" @default.
- NCIT_C176855 hasExactSynonym "BB305 Transduced SCD CD34+ HSCs bb1111" @default.
- NCIT_C176855 hasExactSynonym "LOVOTIBEGLOGENE AUTOTEMCEL" @default.
- NCIT_C176855 hasExactSynonym "LentiGlobin BB305 LVV-transduced Autologous SCD CD34+ HSCs bb1111" @default.
- NCIT_C176855 hasExactSynonym "LentiGlobin BB305 Lentiviral Vector-transduced Autologous SCD CD34+ Hemaptopoietic Stem Cells bb1111" @default.
- NCIT_C176855 hasExactSynonym "LentiGlobin Drug Product for SCD" @default.
- NCIT_C176855 hasExactSynonym "LentiGlobin Drug Product for Sickle Cell Disease" @default.
- NCIT_C176855 hasExactSynonym "LentiGlobin for SCD bb1111" @default.
- NCIT_C176855 hasExactSynonym "Lovotibeglogene Autotemcel" @default.
- NCIT_C176855 hasExactSynonym "bb 1111" @default.
- NCIT_C176855 hasExactSynonym "bb-1111" @default.
- NCIT_C176855 hasExactSynonym "bb1111" @default.
- NCIT_C176855 inSubset NCIT_C116977 @default.
- NCIT_C176855 inSubset NCIT_C116978 @default.
- NCIT_C176855 inSubset NCIT_C176424 @default.
- NCIT_C176855 inSubset NCIT_C63923 @default.
- NCIT_C176855 type Class @default.
- NCIT_C176855 isDefinedBy ncit.owl @default.
- NCIT_C176855 label "Lovotibeglogene Autotemcel" @default.
- NCIT_C176855 subClassOf NCIT_C142078 @default.
- NCIT_C176855 subClassOf NCIT_C154231 @default.
- NCIT_C176855 subClassOf NCIT_C176855 @default.
- NCIT_C176855 subClassOf NCIT_C1908 @default.
- NCIT_C176855 subClassOf NCIT_C1909 @default.
- NCIT_C176855 subClassOf NCIT_C1962 @default.
- NCIT_C176855 subClassOf NCIT_C28681 @default.
- NCIT_C176855 subClassOf NCIT_C307 @default.
- NCIT_C176855 subClassOf NCIT_C308 @default.