ArticleMolecular genetics and metabolism2014
Standardization of α-L-iduronidase enzyme assay with Michaelis-Menten kinetics.
Article in Molecular genetics and metabolism, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01870375 (Longitudinal Studies of Brain Structure and Function in MPS Disorders), which is not on this map. Cited by 16 papers.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Longitudinal Studies of Brain Structure and Function in MPS Disorders
Who cites it
16 citing papers in PubMed.
- An engineered U7 small nuclear RNA scaffold greatly increases ADAR-mediated programmable RNA base editing.Nature communications · 2025Article
- Antibodies to recombinant human alpha-L-iduronidase prevent disease correction in cortical bone in MPS I mice.Molecular therapy. Methods & clinical development · 2025Article
- Ready-to-use iPSC-derived microglia progenitors for the treatment of CNS disease in mouse models of neuropathic mucopolysaccharidoses.Nature communications · 2024Article
- Inhibitors of dermatan sulfate epimerase 1 decreased accumulation of glycosaminoglycans in mucopolysaccharidosis type I fibroblasts.Glycobiology · 2024Article
- Synthesis of Os@ZIF-8 nanocomposites with enhanced peroxidase-like activity for detection of HgRSC advances · 2024Article
- Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system.Molecular genetics and metabolism reports · 2022Article
- Inhibition of iduronic acid biosynthesis by ebselen reduces glycosaminoglycan accumulation in mucopolysaccharidosis type I fibroblasts.Glycobiology · 2021Article
- In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease.Nature communications · 2021Article
- Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement.Biomolecules · 2021Review
- A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Emerging Approaches for Fluorescence-Based Newborn Screening of Mucopolysaccharidoses.Diagnostics (Basel, Switzerland) · 2020Review
- Biochemical diagnosis of mucopolysaccharidosis in a Mexican reference center.Genetics and molecular biology · 2020Article
- Brassica rapa hairy root based expression system leads to the production of highly homogenous and reproducible profiles of recombinant human alpha-L-iduronidase.Plant biotechnology journal · 2019Article
- ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- RTB lectin-mediated delivery of lysosomal α-l-iduronidase mitigates disease manifestations systemically including the central nervous system.Molecular genetics and metabolism · 2018Article
- Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I.Molecular genetics and metabolism reports · 2016Article
Corrections and comments
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Authors and funding
4 authors.
Funding
Abstract
The lack of methodological uniformity in enzyme assays has been a long-standing difficulty, a problem for bench researchers, for the interpretation of clinical diagnostic tests, and an issue for investigational drug review. Illustrative of the problem, α-L-iduronidase enzyme catalytic activity is frequently measured with the substrate 4-methylumbelliferyl-α-L-iduronide (4MU-iduronide); however, final substrate concentrations used in different assays vary greatly, ranging from 25 μM to 1425 μM (Km ≈ 180 μM) making it difficult to compare results between laboratories. In this study, α-L-iduronidase was assayed with 15 different substrate concentrations. The resulting activity levels from the same specimens varied greatly with different substrate concentrations but, as a group, obeyed the expectations of Michaelis-Menten kinetics. Therefore, for the sake of improved comparability, it is proposed that α-L-iduronidase enzyme assays should be conducted either (1) under substrate saturating conditions; or (2) when concentrations are significantly below substrate saturation, with results standardized by arithmetic adjustment that considers Michaelis-Menten kinetics. The approach can be generalized to many other enzyme assays.
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Registered trials
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