Preferred Label : Iduronicrin Genleukocel-T;
NCIt synonyms : Autologous Sleeping Beauty Transposon-engineered alpha-L-iduronidase-expressing Plasmablasts;
NCIt definition : A preparation of autologous plasmablasts genetically engineered to express alpha-L-iduronidase
(IDUA; laronidase) using the Sleeping Beauty (SB) transposon system, that may potentially
be used for enzyme replacement therapy for Mucopolysaccharidosis type I (MPS I; Hurler
syndrome). Upon administration, iduronicrin genleukocel-T expresses and replaces IDUA.
IDUA, an enzyme required for the lysosomal degradation of glycosaminoglycans (GAGs),
hydrolyzes the non-reducing terminal alpha-L-iduronic acid residues in GAGs, including
dermatan sulfate and heparan sulfate. IDUA deficiency causes accumulations of heparan
sulfate and dermatan sulfate in various tissues of the body which leads to progressive
damage. Mutations in the IDUA gene are responsible for the deficiency of IDUA.;
Molecule name : ISP-001; ISP 001;
NCI Metathesaurus CUI : CL1906518;
Origin ID : C199612;
UMLS CUI : C5855712;
Semantic type(s)
- Cell [UMLS semantic type]
- concept_is_in_subset
