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Target intelligence / Profile preview
Alpha-L-iduronidase (IDUA)
- Target
- IDUA
- Molecular classification
- Enzyme, Glycosidase, Lysosomal enzyme
01
Overview
Alpha-L-iduronidase is a lysosomal enzyme that hydrolyzes terminal α-L-iduronic acid residues from dermatan sulfate and heparan sulfate. Deficiency leads to mucopolysaccharidosis type I (MPS I), a lysosomal storage disorder characterized by GAG accumulation. Recombinant human alpha-L-iduronidase (laronidase) is used as enzyme replacement therapy. Diagnosis involves enzymatic assays and genetic testing.
- Other names
- L-iduronidaseα-L-iduronidaselaronidaseGlycosaminoglycan alpha-L-iduronohydrolase
02
Mechanism of action
Enzyme replacement therapy
03
Biological functions
Glycosaminoglycan catabolismHydrolysis of dermatan sulfateHydrolysis of heparan sulfateLysosomal degradation
04
Disease associations
Mucopolysaccharidosis type IHurler syndromeScheie syndromeHurler-Scheie syndromeLysosomal storage disorder
05
Safety considerations
Infusion-related reactions with laronidaseAntibody development against laronidase
06
Interacting drugs
Laronidase (Aldurazyme)
07
Biomarkers
Alpha-L-iduronidase activity in leukocytes or fibroblastsIDUA gene mutations
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