Preferred Label : Mozafancogene Autotemcel;
NCIt synonyms : Autologous CD34 -enriched Hematopoietic Stem and Progenitor Cells Transduced with
Lentiviral Vector Carrying FANCA Gene RP-L102; Autologous CD34 HSPCs Transduced with LV Carrying FANCA Gene RP-L102; Autologous CD34 -enriched HSPCs Transduced with Lentiviral Vector Carrying FANCA Gene
RP-L102;
NCIt definition : A preparation of autologous CD34 -enriched hematopoietic stem and progenitor cells
(HSPCs) from mobilized peripheral blood of patients with Fanconi anemia subtype A
(FA-A) that are transduced ex vivo with a lentiviral vector carrying the FANCA gene,
with potential to restore FANCA expression and function. Upon re-infusion of mozafancogene
autotemcel back into the patient, these cells express functional FANCA gene. Mutations
in the FANCA gene, common in patients with FA, prevent normal DNA repair which may
lead to chromosomal breakage, increased sensitivity to oxidative and environmental
stress, and bone marrow failure.;
UNII : D82H29W72K;
Molecule name : RP-L102; RP L102;
NCI Metathesaurus CUI : CL1407337;
Origin ID : C173602;
UMLS CUI : C5418006;
Semantic type(s)
- Cell [UMLS semantic type]
- concept_is_in_subset
