Preferred Label : Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive
Hematopoietic Progenitor Cells;
NCIt synonyms : Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive
HPCs;
NCIt definition : Autologous, CD34-positive hematopoietic progenitor cells (HPCs) transduced with a
lentiviral vector encoding three anti-human immunodeficiency virus (HIV) genes: a
short hairpin RNA (shRNA) that targets human chemokine receptor 5 (CCR5), a human/rhesus
macaque chimeric tripartite motif-containing protein 5 alpha isoform (TRIM5alpha),
and a TAT activation response (TAR) decoy, as well as a pre-selective cell-surface
marker, which is a truncated and mutated form of human CD25, used to potentially provide
resistance against human immunodeficiency virus (HIV) infection. Human autologous
CD34-positive HPCs are isolated and transduced ex vivo with the lentiviral vector.
Upon pre-selection, purification using CD25 immunomagnetic separation, and subsequent
administration of effectively transduced HPCs, the HPCs display 3 separate mechanisms
of action against HIV infection: CCR5 shRNA binds to CCR5 mRNA and inhibits the expression
of CCR5, a HIV-1 co-receptor that mediates HIV attachment and host cell entry; TRIM5alpha
prevents HIV genome integration upon cell entry; and the TAR decoy binds to the HIV
TAT protein and prevents TAT-dependent viral gene transcription, thereby preventing
HIV replication. Upon transfer of the lentivirus vector CCR5 shRNA/TRIM5alpha/TAR
decoy-transduced autologous CD34-positive HPCs into the patient, the HPCs are resistant
to HIV entry and replication and could provide long-term protection against both HIV
infection and HIV-associated cancers.;
NCI Metathesaurus CUI : CL498294;
Origin ID : C123931;
UMLS CUI : C4086542;
Semantic type(s)
- Cell [UMLS semantic type]
- concept_is_in_subset
