Literature DB >> 24332804

Standardization of α-L-iduronidase enzyme assay with Michaelis-Menten kinetics.

Li Ou1, Tyler L Herzog2, Carrie M Wilmot3, Chester B Whitley4.   

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.
Copyright © 2013 Elsevier Inc. All rights reserved.

Entities:  

Keywords:  4-methylumbelliferyl; Hurler syndrome; IDUA; Lineweaver–Burk plot; MPS; MU; Michaelis–Menten kinetics; mucopolysaccharidoses; mucopolysaccharidosis; α-L-iduronidase; α-L-iduronidase enzyme assay

Mesh:

Substances:

Year:  2013        PMID: 24332804      PMCID: PMC4014300          DOI: 10.1016/j.ymgme.2013.11.009

Source DB:  PubMed          Journal:  Mol Genet Metab        ISSN: 1096-7192            Impact factor:   4.797


  17 in total

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Authors:  C B Whitley; N K Ramsay; J H Kersey; W Krivit
Journal:  Birth Defects Orig Artic Ser       Date:  1986

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Authors:  C B Whitley; R J Gorlin; W Krivit
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3.  Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses.

Authors:  Elena L Aronovich; Jason B Bell; Lalitha R Belur; Roland Gunther; Brenda Koniar; David C C Erickson; Patricia A Schachern; Ilze Matise; R Scott McIvor; Chester B Whitley; Perry B Hackett
Journal:  J Gene Med       Date:  2007-05       Impact factor: 4.565

4.  Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice.

Authors:  Yuli Liu; Lingfei Xu; Anne K Hennig; Attila Kovacs; Annabel Fu; Sarah Chung; David Lee; Bin Wang; Ramin S Herati; Judith Mosinger Ogilvie; Shi-Rong Cai; Katherine Parker Ponder
Journal:  Mol Ther       Date:  2005-01       Impact factor: 11.454

5.  Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

Authors:  Xiucui Ma; Yuli Liu; Mindy Tittiger; Anne Hennig; Attila Kovacs; Sarah Popelka; Baomei Wang; Ramin Herati; Mark Bigg; Katherine P Ponder
Journal:  Mol Ther       Date:  2007-02-20       Impact factor: 11.454

6.  Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene.

Authors:  Seth D Hartung; Joel L Frandsen; Dao Pan; Brenda L Koniar; Patrick Graupman; Roland Gunther; Walter C Low; Chester B Whitley; R Scott McIvor
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7.  Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy.

Authors:  Mayra F Garcia-Rivera; Leah E Colvin-Wanshura; Matthew S Nelson; Zhenhong Nan; Shaukat A Khan; Tyson B Rogers; Indrani Maitra; Walter C Low; Pankaj Gupta
Journal:  Brain Res Bull       Date:  2007-08-06       Impact factor: 4.077

8.  Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow.

Authors:  Yi Zheng; Nora Rozengurt; Sergey Ryazantsev; Donald B Kohn; Noriko Satake; Elizabeth F Neufeld
Journal:  Mol Genet Metab       Date:  2003-08       Impact factor: 4.797

9.  Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice.

Authors:  Ramin Sedaghat Herati; Xiucui Ma; Mindy Tittiger; Kevin K Ohlemiller; Attila Kovacs; Katherine P Ponder
Journal:  J Gene Med       Date:  2008-09       Impact factor: 4.565

10.  Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.

Authors:  Carmela Di Domenico; Guglielmo R D Villani; Daniele Di Napoli; Enrico Gonzalez Y Reyero; Angelo Lombardo; Luigi Naldini; Paola Di Natale
Journal:  Hum Gene Ther       Date:  2005-01       Impact factor: 5.695

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  10 in total

1.  A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I.

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Journal:  Mol Ther       Date:  2020-04-08       Impact factor: 11.454

2.  RTB lectin-mediated delivery of lysosomal α-l-iduronidase mitigates disease manifestations systemically including the central nervous system.

Authors:  Li Ou; Michael J Przybilla; Brenda Koniar; Chester B Whitley
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3.  Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I.

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Review 4.  Emerging Approaches for Fluorescence-Based Newborn Screening of Mucopolysaccharidoses.

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5.  Brassica rapa hairy root based expression system leads to the production of highly homogenous and reproducible profiles of recombinant human alpha-L-iduronidase.

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Journal:  Plant Biotechnol J       Date:  2018-08-30       Impact factor: 9.803

6.  ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.

Authors:  Li Ou; Russell C DeKelver; Michelle Rohde; Susan Tom; Robert Radeke; Susan J St Martin; Yolanda Santiago; Scott Sproul; Michael J Przybilla; Brenda L Koniar; Kelly M Podetz-Pedersen; Kanut Laoharawee; Renee D Cooksley; Kathleen E Meyer; Michael C Holmes; R Scott McIvor; Thomas Wechsler; Chester B Whitley
Journal:  Mol Ther       Date:  2018-11-01       Impact factor: 12.910

Review 7.  Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement.

Authors:  Christiane S Hampe; Jacob Wesley; Troy C Lund; Paul J Orchard; Lynda E Polgreen; Julie B Eisengart; Linda K McLoon; Sebahattin Cureoglu; Patricia Schachern; R Scott McIvor
Journal:  Biomolecules       Date:  2021-01-29

8.  Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system.

Authors:  Sarah Kim; Michael J Przybilla; Chester B Whitley; Li Ou; Mahmoud Al-Kofahi; Jeanine R Jarnes
Journal:  Mol Genet Metab Rep       Date:  2022-09-18

9.  In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease.

Authors:  Sourav K Bose; Brandon M White; Meghana V Kashyap; Apeksha Dave; Felix R De Bie; Haiying Li; Kshitiz Singh; Pallavi Menon; Tiankun Wang; Shiva Teerdhala; Vishal Swaminathan; Heather A Hartman; Sowmya Jayachandran; Prashant Chandrasekaran; Kiran Musunuru; Rajan Jain; David B Frank; Philip Zoltick; William H Peranteau
Journal:  Nat Commun       Date:  2021-07-13       Impact factor: 14.919

10.  Inhibition of iduronic acid biosynthesis by ebselen reduces glycosaminoglycan accumulation in mucopolysaccharidosis type I fibroblasts.

Authors:  Marco Maccarana; Emil Tykesson; Edgar M Pera; Nadège Gouignard; Jianping Fang; Anders Malmström; Giancarlo Ghiselli; Jin-Ping Li
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  10 in total

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