Preferred Label : Autologous CD34 -enriched HSPCs Transduced with LV Vector Carrying TCIRG1 Gene RP-L401;
NCIt synonyms : Autologous CD34 HSPCs Transduced with LV Vector Encoding TCIRG1 Gene RP-L401; Autologous CD34 -enriched Hematopoietic Stem and Progenitor Cells Transduced with
Lentiviral Vector Carrying TCIRG1 Gene RP-L401; Autologous CD34 -enriched HSPCs-expressing TCIRG1 Gene RP-L401;
NCIt definition : A preparation of autologous CD34 -enriched hematopoietic stem and progenitor cells
(HSPCs) from mobilized peripheral blood of patients with infantile malignant osteopetrosis
(IMO) that are transduced ex vivo with a lentiviral (LV) vector carrying the T cell
immune regulator 1 (TCIRG1) gene, with potential to restore TCIRG1 expression and
function. Upon re-infusion of autologous CD34 -enriched HSPCs transduced with LV vector
carrying TCIRG1 gene RP-L401 back into the patient, these cells express functional
TCIRG1 gene. Mutations in the TCIRG1 gene, found in some patients with IMO, lead to
functional impairment of the proton pump of the osteoclast.;
Molecule name : RP L401; RP-L401;
NCI Metathesaurus CUI : CL1773738;
Origin ID : C185445;
UMLS CUI : C5667000;
Semantic type(s)
- Cell [UMLS semantic type]
- concept_is_in_subset
