Low catalytic activity is insufficient to induce disease pathology in triosephosphate isomerase deficiency.


Journal

Journal of inherited metabolic disease
ISSN: 1573-2665
Titre abrégé: J Inherit Metab Dis
Pays: United States
ID NLM: 7910918

Informations de publication

Date de publication:
09 2019
Historique:
received: 26 10 2018
revised: 11 04 2019
accepted: 24 04 2019
pubmed: 22 5 2019
medline: 17 9 2020
entrez: 22 5 2019
Statut: ppublish

Résumé

Triosephosphate isomerase (TPI) deficiency is a fatal genetic disorder characterized by hemolytic anemia and neurological dysfunction. Although the enzyme defect in TPI was discovered in the 1960s, the exact etiology of the disease is still debated. Some aspects indicate the disease could be caused by insufficient enzyme activity, whereas other observations indicate it could be a protein misfolding disease with tissue-specific differences in TPI activity. We generated a mouse model in which exchange of a conserved catalytic amino acid residue (isoleucine to valine, Ile170Val) reduces TPI specific activity without affecting the stability of the protein dimer. TPI

Identifiants

pubmed: 31111503
doi: 10.1002/jimd.12105
pmc: PMC7887927
doi:

Substances chimiques

Triose-Phosphate Isomerase EC 5.3.1.1

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

839-849

Subventions

Organisme : Wellcome Trust
Pays : United Kingdom
Organisme : Cancer Research UK
ID : FC001134
Pays : United Kingdom
Organisme : Medical Research Council
ID : FC001134
Pays : United Kingdom
Organisme : Wellcome Trust
ID : FC001134
Pays : United Kingdom

Informations de copyright

© 2019 The Authors. Journal of Inherited Metabolic Disease published by John Wiley & Sons Ltd on behalf of SSIEM.

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Auteurs

Joanna Segal (J)

The Molecular Biology of Metabolism Laboratory, Francis Crick Institute, London, UK.

Michael Mülleder (M)

The Molecular Biology of Metabolism Laboratory, Francis Crick Institute, London, UK.
Max Planck Institute for Molecular Genetics, Berlin, Germany.

Antje Krüger (A)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Thure Adler (T)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Institute for Medical Microbiology, Immunology, and Hygiene, Munich, Germany.

Manuela Scholze-Wittler (M)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Lore Becker (L)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-Universität München, Munich, Germany.

Julia Calzada-Wack (J)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Institute of Pathology, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.

Lillian Garrett (L)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Institute of Developmental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.

Sabine M Hölter (SM)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Institute of Developmental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.

Birgit Rathkolb (B)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Chair for Molecular Animal Breeding and Biotechnology, Gene Center, Ludwig-Maximilians-Universität München, Munich, Germany.
Member of German Center for Diabetes Research (DZD), Neuherberg/Munich, Germany.

Jan Rozman (J)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Member of German Center for Diabetes Research (DZD), Neuherberg/Munich, Germany.
Molecular Nutritional Medicine, Else Kröner-Fresenius Center, TUM, Freising-Weihenstephan, Germany.

Ildiko Racz (I)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.

Ralf Fischer (R)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.

Dirk H Busch (DH)

Institute for Medical Microbiology, Immunology, and Hygiene, Munich, Germany.

Frauke Neff (F)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Institute of Pathology, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.

Martin Klingenspor (M)

Molecular Nutritional Medicine, Else Kröner-Fresenius Center, TUM, Freising-Weihenstephan, Germany.
ZIEL - Institute for Food and Health, Technical University Munich, Freising-Weihenstephan, Germany.

Thomas Klopstock (T)

Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-Universität München, Munich, Germany.
Munich Cluster for Systems Neurology (SyNergy), Adolf-Butenandt-Institut, Ludwig-Maximilians-Universität München, Munich, Germany.
Deutsches Zentrum für Neurodegenerative Erkrankungen e. V. (DZNE) Site Munich, Munich, Germany.

Nana-Maria Grüning (NM)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Steve Michel (S)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Beata Lukaszewska-McGreal (B)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Ingo Voigt (I)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Ludger Hartmann (L)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Bernd Timmermann (B)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Hans Lehrach (H)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Eckhard Wolf (E)

Chair for Molecular Animal Breeding and Biotechnology, Gene Center, Ludwig-Maximilians-Universität München, Munich, Germany.

Wolfgang Wurst (W)

Institute of Developmental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Munich Cluster for Systems Neurology (SyNergy), Adolf-Butenandt-Institut, Ludwig-Maximilians-Universität München, Munich, Germany.
Deutsches Zentrum für Neurodegenerative Erkrankungen e. V. (DZNE) Site Munich, Munich, Germany.
Chair of Developmental Genetics, TUM, Freising-Weihenstephan, Germany.

Valérie Gailus-Durner (V)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.

Helmut Fuchs (H)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.

Martin H de Angelis (M)

German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, German Research Center for Environmental Health (GmbH), Neuherberg/Munich, Germany.
Member of German Center for Diabetes Research (DZD), Neuherberg/Munich, Germany.
Chair of Experimental Genetics, Center of Life and Food Sciences Weihenstephan, TUM, Freising-Weihenstephan, Germany.

Heinrich Schrewe (H)

Max Planck Institute for Molecular Genetics, Berlin, Germany.

Mariia Yuneva (M)

Oncogenes and Tumour Metabolism Laboratory, The Francis Crick Institute, London, UK.

Markus Ralser (M)

The Molecular Biology of Metabolism Laboratory, Francis Crick Institute, London, UK.
Max Planck Institute for Molecular Genetics, Berlin, Germany.
Cambridge Systems Biology Centre and Department of Biochemistry, University of Cambridge, Cambridge, UK.
Department of Biochemistry, Charitè Universitätsmedizin Berlin, Berlin, Germany.

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