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Version: 17.06d (Release date: 2017-06-26)
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Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells (Code C111902)

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Terms & Properties

Preferred Name: Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells

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.

Display Name: Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells

Label: Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells

NCI Thesaurus Code: C111902 (Search for linked caDSR metadata)  (search value sets)

NCI Metathesaurus Link: CL454234  (see NCI Metathesaurus info)

Synonyms & Abbreviations: (see Synonym Details)
Lenti-D/ABCD1-transduced Autologous Hematopoietic Stem Cells

External Source Codes: 
PDQ Closed Trial Search ID 753681
PDQ Open Trial Search ID 753681 (check for NCI PDQ open clinical trial info)
NCI META CUI CL454234

Other Properties:
     Name Value (qualifiers indented underneath)
code C111902
Contributing_Source CTRP
Semantic_Type Pharmacologic Substance

Additional Concept Data: 
Defined Fully by Roles: No  

URL: https://ncit.nci.nih.gov/ncitbrowser/ConceptReport.jsp?dictionary=NCI_Thesaurus&code=C111902

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