Preferred Label : Autologous CD34 enriched Alpha-L-iduronidase-expressing Hematopoietic Stem Cells
OTL-203;
NCIt synonyms : Autologous CD34 enriched IDUA-expressing HSCs OTL-203; Autologous IDUA-expressing HSCs OTL-203;
NCIt definition : A gene therapy agent composed of a preparation of autologous CD34-enriched hematopoietic
stem cells (HSCs) transduced with a lentiviral vector expressing the endogenous human
gene alpha-L-iduronidase (IDUA), that may potentially be used in the treatment of
mucopolysaccharidosis type I (MPS I; Hurler syndrome). Upon administration, autologous
CD34 enriched IDUA-expressing HSCs OTL-203 express IDUA, which is an enzyme required
for the lysosomal degradation of glycosaminoglycans (GAGs). IDUA 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 organs of the body which leads to progressive damage. Mutations in the
IDUA gene are responsible for the deficiency of IDUA.;
Molecule name : OTL-203; OTL 203;
NCI Metathesaurus CUI : CL1927034;
Origin ID : C204810;
UMLS CUI : C5853562;
Semantic type(s)
- Cell [UMLS semantic type]
- concept_is_in_subset
