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Target intelligence / Profile preview
Iduronidase (IDUA)
- Target
- IDUA
- Molecular classification
- Enzyme, Glycosidase, Lysosomal enzyme, Hydrolase
01
Overview
Iduronidase, also known as alpha-L-iduronidase, is a lysosomal enzyme responsible for the degradation of glycosaminoglycans (GAGs), specifically heparan sulfate and dermatan sulfate. Deficiency leads to Mucopolysaccharidosis Type I (MPS I). Treatment includes enzyme replacement therapy and hematopoietic cell transplantation.
- Other names
- Alpha-L-iduronidase
02
Mechanism of action
Enzyme replacement therapy (ERT) to provide functional iduronidase for GAG breakdown.
03
Biological functions
Glycosaminoglycan (GAG) degradationHydrolysis of α-L-iduronic acid residuesLysosomal catabolism
04
Disease associations
Mucopolysaccharidosis Type I (MPS I)Lysosomal storage disorder
05
Safety considerations
ERT efficacy limited by blood-brain barrierNeurological manifestations of MPS I may not be fully addressed by ERTEnzyme replacement therapy infusion reactions
06
Interacting drugs
Laronidase (Aldurazyme)
07
Biomarkers
Alpha-L-iduronidase activity levelsHeparan sulfate levelsDermatan sulfate levelsIDUA mutations
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