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- NCIT_C111902 IAO_0000115 "Autologous CD34+ hematopoietic stem cells transduced with Lenti-D lentiviral vector encoding the human ATP-binding cassette, sub-family D, member 1 (ABCD1) cDNA, for the potential treatment of childhood cerebral adrenoleukodystrophy (CCALD). Upon administration of the lenti-D/ABCD1-transduced autologous hematopoietic stem cells, the cells proliferate, and some travel to the brain and differentiate into microglial cells. The expressed ABCD1 restores the function of ALD protein (ALDP) and aids in the treatment of CCALD. In CCALD, the ABCD1 gene is mutated, leading to the inability of patients to metabolize very long chain fatty acids in cells of the brain." @default.
- NCIT_C111902 NCIT_NHC0 "C111902" @default.
- NCIT_C111902 NCIT_P106 "Pharmacologic Substance" @default.
- NCIT_C111902 NCIT_P107 "Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells" @default.
- NCIT_C111902 NCIT_P108 "Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells" @default.
- NCIT_C111902 NCIT_P208 "CL454234" @default.
- NCIT_C111902 NCIT_P322 "CTRP" @default.
- NCIT_C111902 NCIT_P329 "753681" @default.
- NCIT_C111902 NCIT_P330 "753681" @default.
- NCIT_C111902 NCIT_P399 "753681" @default.
- NCIT_C111902 normalizedInformationContent "100" @default.
- NCIT_C111902 referenceCount "1" @default.
- NCIT_C111902 hasExactSynonym "Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells" @default.
- NCIT_C111902 inSubset NCIT_C116977 @default.
- NCIT_C111902 inSubset NCIT_C116978 @default.
- NCIT_C111902 inSubset NCIT_C176424 @default.
- NCIT_C111902 type Class @default.
- NCIT_C111902 isDefinedBy ncit.owl @default.
- NCIT_C111902 label "Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells" @default.
- NCIT_C111902 subClassOf NCIT_C111902 @default.
- NCIT_C111902 subClassOf NCIT_C142078 @default.
- NCIT_C111902 subClassOf NCIT_C1908 @default.
- NCIT_C111902 subClassOf NCIT_C1909 @default.
- NCIT_C111902 subClassOf NCIT_C201554 @default.
- NCIT_C111902 subClassOf NCIT_C307 @default.
- NCIT_C111902 subClassOf NCIT_C308 @default.