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- NCIT_C171808 IAO_0000115 "A gene replacement therapy agent composed of autologous cluster of differentiation 34 (CD34)-positive human hematopoietic stem cells (HSCs) transduced ex vivo with Lenti-D lentiviral vector (LVV), a replication-incompetent, self-inactivating LVV carrying ABCD1 complementary DNA (cDNA) that encodes normal adrenoleukodystrophy protein (ALD protein; ALDP), that can be used to treat early cerebral adrenoleukodystrophy (CALD). Upon administration, elivaldogene autotemcel engrafts in the bone marrow and differentiates into various cell types, including monocytes, that produce functional copies of the ABCD1 cDNA and thus functional ALDP. Functional ALDP degrades very long chain fatty acids (VLCFAs), which prevents accumulation of VLCFAs and may decrease or prevent further inflammation, demyelination and nerve damage. This slows the progression of neurologic dysfunction in patients with early, active cerebral adrenoleukodystrophy (CALD)." @default.
- NCIT_C171808 NCIT_NHC0 "C171808" @default.
- NCIT_C171808 NCIT_P106 "Pharmacologic Substance" @default.
- NCIT_C171808 NCIT_P108 "Elivaldogene Autotemcel" @default.
- NCIT_C171808 NCIT_P208 "CL1406003" @default.
- NCIT_C171808 NCIT_P319 "KUM75TD6SG" @default.
- NCIT_C171808 NCIT_P322 "FDA" @default.
- NCIT_C171808 normalizedInformationContent "100" @default.
- NCIT_C171808 referenceCount "1" @default.
- NCIT_C171808 hasExactSynonym "ELIVALDOGENE AUTOTEMCEL" @default.
- NCIT_C171808 hasExactSynonym "Eli-cel" @default.
- NCIT_C171808 hasExactSynonym "Elivaldogene Autotemcel" @default.
- NCIT_C171808 hasExactSynonym "Lenti-D (TM)" @default.
- NCIT_C171808 hasExactSynonym "Skysona" @default.
- NCIT_C171808 inSubset NCIT_C63923 @default.
- NCIT_C171808 type Class @default.
- NCIT_C171808 isDefinedBy ncit.owl @default.
- NCIT_C171808 label "Elivaldogene Autotemcel" @default.
- NCIT_C171808 subClassOf NCIT_C142078 @default.
- NCIT_C171808 subClassOf NCIT_C154231 @default.
- NCIT_C171808 subClassOf NCIT_C171808 @default.
- NCIT_C171808 subClassOf NCIT_C1908 @default.
- NCIT_C171808 subClassOf NCIT_C1909 @default.
- NCIT_C171808 subClassOf NCIT_C1962 @default.
- NCIT_C171808 subClassOf NCIT_C307 @default.
- NCIT_C171808 subClassOf NCIT_C308 @default.