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- NCIT_C173602 IAO_0000115 "A preparation of autologous CD34+-enriched hematopoietic stem and progenitor cells (HSPCs) from mobilized peripheral blood of patients with Fanconi anemia subtype A (FA-A) that are transduced ex vivo with a lentiviral vector carrying the FANCA gene, with potential to restore FANCA expression and function. Upon re-infusion of mozafancogene autotemcel back into the patient, these cells express functional FANCA gene. Mutations in the FANCA gene, common in patients with FA, prevent normal DNA repair which may lead to chromosomal breakage, increased sensitivity to oxidative and environmental stress, and bone marrow failure." @default.
- NCIT_C173602 NCIT_NHC0 "C173602" @default.
- NCIT_C173602 NCIT_P106 "Pharmacologic Substance" @default.
- NCIT_C173602 NCIT_P107 "Mozafancogene Autotemcel" @default.
- NCIT_C173602 NCIT_P108 "Mozafancogene Autotemcel" @default.
- NCIT_C173602 NCIT_P208 "CL1407337" @default.
- NCIT_C173602 NCIT_P319 "D82H29W72K" @default.
- NCIT_C173602 NCIT_P322 "CTRP" @default.
- NCIT_C173602 NCIT_P322 "FDA" @default.
- NCIT_C173602 NCIT_P329 "802240" @default.
- NCIT_C173602 NCIT_P330 "802240" @default.
- NCIT_C173602 NCIT_P399 "802240" @default.
- NCIT_C173602 normalizedInformationContent "100" @default.
- NCIT_C173602 referenceCount "1" @default.
- NCIT_C173602 hasExactSynonym "Autologous CD34+ HSPCs Transduced with LV Carrying FANCA Gene RP-L102" @default.
- NCIT_C173602 hasExactSynonym "Autologous CD34+-enriched HSPCs Transduced with Lentiviral Vector Carrying FANCA Gene RP-L102" @default.
- NCIT_C173602 hasExactSynonym "Autologous CD34+-enriched Hematopoietic Stem and Progenitor Cells Transduced with Lentiviral Vector Carrying FANCA Gene RP-L102" @default.
- NCIT_C173602 hasExactSynonym "MOZAFANCOGENE AUTOTEMCEL" @default.
- NCIT_C173602 hasExactSynonym "Mozafancogene Autotemcel" @default.
- NCIT_C173602 hasExactSynonym "RP L102" @default.
- NCIT_C173602 hasExactSynonym "RP-L102" @default.
- NCIT_C173602 hasExactSynonym "RPL102" @default.
- NCIT_C173602 inSubset NCIT_C116977 @default.
- NCIT_C173602 inSubset NCIT_C116978 @default.
- NCIT_C173602 inSubset NCIT_C176424 @default.
- NCIT_C173602 inSubset NCIT_C63923 @default.
- NCIT_C173602 type Class @default.
- NCIT_C173602 isDefinedBy ncit.owl @default.
- NCIT_C173602 label "Mozafancogene Autotemcel" @default.
- NCIT_C173602 subClassOf NCIT_C142078 @default.
- NCIT_C173602 subClassOf NCIT_C173602 @default.
- NCIT_C173602 subClassOf NCIT_C1908 @default.
- NCIT_C173602 subClassOf NCIT_C1909 @default.
- NCIT_C173602 subClassOf NCIT_C201554 @default.
- NCIT_C173602 subClassOf NCIT_C307 @default.
- NCIT_C173602 subClassOf NCIT_C308 @default.