MINPP1 prevents intracellular accumulation of the chelator inositol hexakisphosphate and is mutated in Pontocerebellar Hypoplasia.


Journal

Nature communications
ISSN: 2041-1723
Titre abrégé: Nat Commun
Pays: England
ID NLM: 101528555

Informations de publication

Date de publication:
30 11 2020
Historique:
received: 29 06 2019
accepted: 29 10 2020
entrez: 1 12 2020
pubmed: 2 12 2020
medline: 22 12 2020
Statut: epublish

Résumé

Inositol polyphosphates are vital metabolic and secondary messengers, involved in diverse cellular functions. Therefore, tight regulation of inositol polyphosphate metabolism is essential for proper cell physiology. Here, we describe an early-onset neurodegenerative syndrome caused by loss-of-function mutations in the multiple inositol-polyphosphate phosphatase 1 gene (MINPP1). Patients are found to have a distinct type of Pontocerebellar Hypoplasia with typical basal ganglia involvement on neuroimaging. We find that patient-derived and genome edited MINPP1

Identifiants

pubmed: 33257696
doi: 10.1038/s41467-020-19919-y
pii: 10.1038/s41467-020-19919-y
pmc: PMC7705663
doi:

Substances chimiques

Chelating Agents 0
Phytic Acid 7IGF0S7R8I
Phosphoric Monoester Hydrolases EC 3.1.3.2
multiple inositol-polyphosphate phosphatase EC 3.1.3.62

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

6087

Subventions

Organisme : Medical Research Council
ID : MR/T028904/1
Pays : United Kingdom

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Auteurs

Ekin Ucuncu (E)

Université de Paris, Developmental Brain Disorders Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Karthyayani Rajamani (K)

Université de Paris, Developmental Brain Disorders Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Miranda S C Wilson (MSC)

MRC Laboratory for Molecular Cell Biology, University College London, WC1E 6BT, London, UK.

Daniel Medina-Cano (D)

Université de Paris, Developmental Brain Disorders Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Nami Altin (N)

Université de Paris, Developmental Brain Disorders Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Pierre David (P)

Transgenesis Platform, Laboratoire d'Expérimentation Animale et Transgenèse (LEAT), Imagine Institute, Structure Fédérative de Recherche Necker INSERM US24/CNRS UMS3633, 75015, Paris, France.

Giulia Barcia (G)

Université de Paris, Developmental Brain Disorders Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.
Département de Génétique Médicale, AP-HP, Hôpital Necker-Enfants Malades, F-75015, Paris, France.

Nathalie Lefort (N)

Université de Paris, iPSC Core Facility, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Céline Banal (C)

Université de Paris, iPSC Core Facility, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Marie-Thérèse Vasilache-Dangles (MT)

Département de Neurologie Pédiatrique, AP-HP, Hôpital Necker-Enfants Malades, F-75015, Paris, France.

Gaële Pitelet (G)

Service de Neuropédiatrie, CHU Nice, 06200, Nice, France.

Elsa Lorino (E)

ESEAN, 44200 Nantes, Service de maladies chroniques de l'enfant, CHU Nantes, 44093, Nantes, France.

Nathalie Rabasse (N)

Service de pédiatrie, hôpital d'Antibes-Juan-les-Pins, 06600, Antibes-Juan-les-Pins, France.

Eric Bieth (E)

Service de Génétique Médicale, CHU Toulouse, 31059, Toulouse, France.

Maha S Zaki (MS)

Clinical Genetics Department, Human Genetics and Genome Research Division, National Research Centre, Cairo, 12311, Egypt.

Meral Topcu (M)

Department of Child Neurology, Faculty of Medicine, Hacettepe University, Ankara, 06100, Turkey.

Fatma Mujgan Sonmez (FM)

Guven Hospital, Child Neurology Department, Ankara, Turkey.
Department of Child Neurology, Faculty of Medicine, Karadeniz Technical University, Trabzon, 61080, Turkey.

Damir Musaev (D)

Laboratory for Pediatric Brain Diseases, Rady Children's Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, 92093, USA.

Valentina Stanley (V)

Laboratory for Pediatric Brain Diseases, Rady Children's Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, 92093, USA.

Christine Bole-Feysot (C)

Université de Paris, Genomics Platform, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Patrick Nitschké (P)

Université de Paris, Bioinformatics Core Facility, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Arnold Munnich (A)

Université de Paris, Translational Genetics Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Nadia Bahi-Buisson (N)

Université de Paris, Genetics and Development of the Cerebral Cortex Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Catherine Fossoud (C)

Centre de Référence des Troubles des Apprentissages, Hôpitaux Pédiatriques de Nice CHU-Lenval, 06200, Nice, France.

Fabienne Giuliano (F)

Service de Génétique Médicale, Centre Hospitalier Universitaire de Nice, 06202, Nice, France.

Laurence Colleaux (L)

Université de Paris, Developmental Brain Disorders Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.

Lydie Burglen (L)

Université de Paris, Developmental Brain Disorders Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France.
Centre de Référence des Malformations et Maladies Congénitales du Cervelet, Département de Génétique, AP-HP, Sorbonne Université, Hôpital Trousseau, 75012, Paris, France.

Joseph G Gleeson (JG)

Laboratory for Pediatric Brain Diseases, Rady Children's Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, 92093, USA.

Nathalie Boddaert (N)

Département de radiologie pédiatrique, INSERM UMR 1163 and INSERM U1000, AP-HP, Hôpital Necker-Enfants Malades, F-75015, Paris, France.

Adolfo Saiardi (A)

MRC Laboratory for Molecular Cell Biology, University College London, WC1E 6BT, London, UK. a.saiardi@ucl.ac.uk.

Vincent Cantagrel (V)

Université de Paris, Developmental Brain Disorders Laboratory, Imagine Institute, INSERM UMR 1163, F-75015, Paris, France. vincent.cantagrel@inserm.fr.

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Classifications MeSH