Preferred Label : Anti-HIV-1 Lentiviral Vector-expressing sh5/C46 Cal-1;
NCIt synonyms : LVsh5/C46; Cal-1 Vector; LVsh5/C46 Vector; Anti-HIV-1 LVsh5/C46; Anti-HIV-1 Lentiviral Vector sh5/C46; Anti-HIV-1 LV-expressing sh5/C46 Cal-1;
NCIt definition : A gene transfer construct composed of a self-inactivating (SIN) lentiviral vector
(LV) expressing a short hairpin RNA (shRNA) that targets the human C-C chemokine receptor
type 5 (CCR5) mRNA (sh5) and expressing the HIV entry inhibitor C46, with potential
anti-human immunodeficiency virus (HIV) type 1 (HIV-1) activity. Upon transduction
of the anti-HIV-1 LVsh5/C46 Cal-1 in specified blood cell populations, such as peripheral
blood mononuclear cells (PBMCs), hematopoietic stem/progenitor cells (HSPCs) and CD4
T-lymphocytes, the cells express shCCR5 and C46. shCCR5 targets and binds to CCR5
mRNA, which inhibits the expression of CCR5 and prevents binding of the virus to the
cellular CCR5 co-receptor. The cell surface expression of the cell membrane-anchored
C46 peptide blocks HIV-1 fusion to the cellular membrane. The removal of CCR5 from
and the production of C46 in the bone marrow and white blood cells, make the transduced
cells resistant to and protect them from HIV-1 entry, infection and replication. HIV-resistant
HSPCs could provide long-term protection against latent HIV infection and against
HIV-associated cancers. C46 is a membrane-anchored 46-amino acid sequence found in
HIV-1 gp41. CCR5 is a HIV-1 co-receptor that mediates HIV attachment and cell entry.;
Molecule name : Cal-1;
NCI Metathesaurus CUI : CL978677;
Origin ID : C165547;
UMLS CUI : C5237257;
Semantic type(s)
- concept_is_in_subset
