Preferred Label : Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells;
NCIt definition : 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.;
NCI Metathesaurus CUI : CL454234;
Origin ID : C111902;
UMLS CUI : C3829297;
Semantic type(s)
- Cell [UMLS semantic type]
- concept_is_in_subset
