Novel <i>DNM1L</i> variants impair mitochondrial dynamics through divergent mechanisms.


Journal

Life science alliance
ISSN: 2575-1077
Titre abrégé: Life Sci Alliance
Pays: United States
ID NLM: 101728869

Informations de publication

Date de publication:
01 08 2022
Historique:
received: 01 11 2021
revised: 07 07 2022
accepted: 07 07 2022
entrez: 1 8 2022
pubmed: 2 8 2022
medline: 4 8 2022
Statut: epublish

Résumé

Imbalances in mitochondrial and peroxisomal dynamics are associated with a spectrum of human neurological disorders. Mitochondrial and peroxisomal fission both involve dynamin-related protein 1 (DRP1) oligomerisation and membrane constriction, although the precise biophysical mechanisms by which distinct DRP1 variants affect the assembly and activity of different DRP1 domains remains largely unexplored. We analysed four unreported de novo heterozygous variants in the dynamin-1-like gene <i>DNM1L</i>, affecting different highly conserved DRP1 domains, leading to developmental delay, seizures, hypotonia, and/or rare cardiac complications in infancy. Single-nucleotide DRP1 stalk domain variants were found to correlate with more severe clinical phenotypes, with in vitro recombinant human DRP1 mutants demonstrating greater impairments in protein oligomerisation, DRP1-peroxisomal recruitment, and both mitochondrial and peroxisomal hyperfusion compared to GTPase or GTPase-effector domain variants. Importantly, we identified a novel mechanism of pathogenesis, where a p.Arg710Gly variant uncouples DRP1 assembly from assembly-stimulated GTP hydrolysis, providing mechanistic insight into how assembly-state information is transmitted to the GTPase domain. Together, these data reveal that discrete, pathological <i>DNM1L</i> variants impair mitochondrial network maintenance by divergent mechanisms.

Identifiants

pubmed: 35914810
pii: 5/12/e202101284
doi: 10.26508/lsa.202101284
pmc: PMC9354038
pii:
doi:

Substances chimiques

Microtubule-Associated Proteins 0
Mitochondrial Proteins 0
GTP Phosphohydrolases EC 3.6.1.-
DNM1L protein, human EC 3.6.5.5
Dynamins EC 3.6.5.5

Banques de données

GENBANK
['NM_012062.5']
PDB
['5WP9', '4BEJ', '3W6P', '3W6O']

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : NCATS NIH HHS
ID : TL1 TR001437
Pays : United States
Organisme : Medical Research Council
ID : MR/S005021/1
Pays : United Kingdom
Organisme : NHLBI NIH HHS
ID : R01 HL128240
Pays : United States
Organisme : Wellcome Trust
ID : 203105/Z/16/Z
Pays : United Kingdom
Organisme : Medical Research Council
ID : G0800674
Pays : United Kingdom
Organisme : NIH HHS
ID : S10 OD025036
Pays : United States
Organisme : NIGMS NIH HHS
ID : R01 GM067180
Pays : United States
Organisme : NIGMS NIH HHS
ID : T32 GM080202
Pays : United States
Organisme : Wellcome Trust
Pays : United Kingdom

Informations de copyright

© 2022 Nolden et al.

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Auteurs

Kelsey A Nolden (KA)

Department of Biochemistry, Medical College of Wisconsin, Milwaukee, WI, USA.

John M Egner (JM)

Department of Biochemistry, Medical College of Wisconsin, Milwaukee, WI, USA.

Jack J Collier (JJ)

Wellcome Centre for Mitochondrial Research, Newcastle University, Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle upon Tyne, UK.
Department of Neurology and Neurosurgery, Montreal Neurological Institute, McGill University, Montreal, Canada.

Oliver M Russell (OM)

Wellcome Centre for Mitochondrial Research, Newcastle University, Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle upon Tyne, UK.

Charlotte L Alston (CL)

Wellcome Centre for Mitochondrial Research, Newcastle University, Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle upon Tyne, UK.
The National Health Service (NHS) Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Megan C Harwig (MC)

Department of Biochemistry, Medical College of Wisconsin, Milwaukee, WI, USA.

Michael E Widlansky (ME)

Department of Medicine, Division of Cardiovascular Medicine and Department of Pharmacology, Medical College of Wisconsin, Milwaukee, WI, USA.

Souphatta Sasorith (S)

Laboratoire de Génétique Moléculaire, Centre Hospitalier Universitaire and PhyMedExp, INSERM U1046, CNRS UMR 9214, Montpellier, France.

Inês A Barbosa (IA)

Department of Medical and Molecular Genetics, School of Basic and Medical Biosciences, King's College London, London, UK.

Andrew Gl Douglas (AG)

Wessex Clinical Genetics Service, University Hospital Southampton NHS Foundation Trust, Southampton, UK.
Human Development and Health, Faculty of Medicine, University of Southampton, Southampton, UK.

Julia Baptista (J)

Peninsula Medical School, Faculty of Health, University of Plymouth, Plymouth, UK.

Mark Walker (M)

Department of Cellular Pathology, University Hospital Southampton NHS Foundation Trust, Southampton, UK.

Deirdre E Donnelly (DE)

Northern Ireland Regional Genetics Centre, Belfast Health and Social Care Trust, Belfast City Hospital, Belfast, UK.

Andrew A Morris (AA)

Willink Metabolic Unit, Manchester Centre for Genomic Medicine, Manchester University Hospitals NHS Foundation Trust, Manchester, UK.

Hui Jeen Tan (HJ)

Department of Paediatric Neurology, Royal Manchester Children's Hospital, Manchester University Hospitals NHS Foundation Trust, Manchester, UK.

Manju A Kurian (MA)

Developmental Neurosciences Department, Zayed Centre for Research into Rare Diseases in Children, University College London Great Ormond Street Institute of Child Health, Faculty of Population Health Sciences, London, UK.

Kathleen Gorman (K)

Department of Neurology and Clinical Neurophysiology, Children's Health Ireland at Temple Street, Dublin, Ireland.
School of Medicine and Medical Science, University College Dublin, Dublin, Ireland.

Santosh Mordekar (S)

Department of Paediatric Neurology, Sheffield Children's Hospital, Sheffield, UK.

Charu Deshpande (C)

Clinical Genetics Unit, Guys and St. Thomas' NHS Foundation Trust, London, UK.

Rajib Samanta (R)

Department of Paediatric Neurology, University Hospitals Leicester NHS Trust, Leicester, UK.

Robert McFarland (R)

Wellcome Centre for Mitochondrial Research, Newcastle University, Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle upon Tyne, UK.
The National Health Service (NHS) Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

R Blake Hill (RB)

Department of Biochemistry, Medical College of Wisconsin, Milwaukee, WI, USA.

Robert W Taylor (RW)

Wellcome Centre for Mitochondrial Research, Newcastle University, Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle upon Tyne, UK.
The National Health Service (NHS) Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Monika Oláhová (M)

Wellcome Centre for Mitochondrial Research, Newcastle University, Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle upon Tyne, UK monika.olahova@ncl.ac.uk.

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