TM6SF2/PNPLA3/MBOAT7 Loss-of-Function Genetic Variants Impact on NAFLD Development and Progression Both in Patients and in In Vitro Models.


Journal

Cellular and molecular gastroenterology and hepatology
ISSN: 2352-345X
Titre abrégé: Cell Mol Gastroenterol Hepatol
Pays: United States
ID NLM: 101648302

Informations de publication

Date de publication:
2022
Historique:
received: 26 02 2021
revised: 16 11 2021
accepted: 16 11 2021
pubmed: 26 11 2021
medline: 5 4 2022
entrez: 25 11 2021
Statut: ppublish

Résumé

The I148M Patatin-like Phospholipase Domain-containing 3 (PNPLA3), the rs641738 in the Membrane bound O-acyltransferase domain containing 7-transmembrane channel-like 4 (MBOAT7-TMC4) locus, and the E167K Transmembrane 6 Superfamily Member 2 (TM6SF2) polymorphisms represent the main predisposing factors to nonalcoholic fatty liver disease (NAFLD) development and progression. We previously generated a full knockout of MBOAT7 in HepG2 cells (MBOAT7 NAFLD patients (n = 1380), of whom 121 had HCC, were stratified with a semiquantitative score ranging from 0 to 3 according to the number of PNPLA3, TM6SF2, and MBOAT7 at-risk variants. TM6SF2 was silenced in HepG2 (TM6SF2 In NAFLD patients, the additive weight of these mutations was associated with liver disease severity and an increased risk of developing HCC. In HepG2 cells, TM6SF2 silencing altered lipid composition and induced the accumulation of microvesicular lipid droplets (LDs), whereas the MBOAT7 The co-presence of the 3 at-risk variants impacts the NAFLD course in both patients and experimental models, affecting LD accumulation, mitochondrial functionality, and metabolic reprogramming toward HCC.

Sections du résumé

BACKGROUND & AIMS
The I148M Patatin-like Phospholipase Domain-containing 3 (PNPLA3), the rs641738 in the Membrane bound O-acyltransferase domain containing 7-transmembrane channel-like 4 (MBOAT7-TMC4) locus, and the E167K Transmembrane 6 Superfamily Member 2 (TM6SF2) polymorphisms represent the main predisposing factors to nonalcoholic fatty liver disease (NAFLD) development and progression. We previously generated a full knockout of MBOAT7 in HepG2 cells (MBOAT7
METHODS
NAFLD patients (n = 1380), of whom 121 had HCC, were stratified with a semiquantitative score ranging from 0 to 3 according to the number of PNPLA3, TM6SF2, and MBOAT7 at-risk variants. TM6SF2 was silenced in HepG2 (TM6SF2
RESULTS
In NAFLD patients, the additive weight of these mutations was associated with liver disease severity and an increased risk of developing HCC. In HepG2 cells, TM6SF2 silencing altered lipid composition and induced the accumulation of microvesicular lipid droplets (LDs), whereas the MBOAT7
CONCLUSIONS
The co-presence of the 3 at-risk variants impacts the NAFLD course in both patients and experimental models, affecting LD accumulation, mitochondrial functionality, and metabolic reprogramming toward HCC.

Identifiants

pubmed: 34823063
pii: S2352-345X(21)00241-1
doi: 10.1016/j.jcmgh.2021.11.007
pmc: PMC8783129
pii:
doi:

Substances chimiques

Membrane Proteins 0
TM6SF2 protein, human 0
Acyltransferases EC 2.3.-
MBOAT7 protein, human EC 2.3.-
Lipase EC 3.1.1.3
adiponutrin, human EC 3.1.1.3
Phospholipases A2, Calcium-Independent EC 3.1.1.4

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

759-788

Informations de copyright

Copyright © 2022 The Authors. Published by Elsevier Inc. All rights reserved.

Auteurs

Miriam Longo (M)

General Medicine and Metabolic Diseases, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy; Department of Clinical Sciences and Community Health, Università degli Studi di Milano, Milano, Italy.

Marica Meroni (M)

General Medicine and Metabolic Diseases, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy.

Erika Paolini (E)

General Medicine and Metabolic Diseases, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy; Department of Pharmacological and Biomolecular Sciences, Università degli Studi di Milano, 20133 Milano, Italy.

Veronica Erconi (V)

General Medicine and Metabolic Diseases, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy.

Fabrizia Carli (F)

Institute of Clinical Physiology, National Research Council (CNR), Pisa, Italy.

Francesco Fortunato (F)

Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.

Dario Ronchi (D)

Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.

Roberto Piciotti (R)

General Medicine and Metabolic Diseases, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.

Silvia Sabatini (S)

Institute of Clinical Physiology, National Research Council (CNR), Pisa, Italy.

Chiara Macchi (C)

Department of Pharmacological and Biomolecular Sciences, Università degli Studi di Milano, 20133 Milano, Italy.

Anna Alisi (A)

Research Unit of Molecular Genetics of Complex Phenotypes, "Bambino Gesù" Children's Hospital IRCCS, Rome, Italy.

Luca Miele (L)

Area Medicina Interna, Gastroenterologia e Oncologia Medica, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy.

Giorgio Soardo (G)

Department of Medical Area (DAME), University of Udine and Italian Liver Foundation, Bldg Q AREA Science Park - Basovizza Campus, Trieste, Italy.

Giacomo Pietro Comi (GP)

Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy; Neuromuscular and Rare Diseases Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.

Luca Valenti (L)

Trasfusional Center-Translational Medicine, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy.

Massimiliano Ruscica (M)

Department of Pharmacological and Biomolecular Sciences, Università degli Studi di Milano, 20133 Milano, Italy.

Anna L Fracanzani (AL)

General Medicine and Metabolic Diseases, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.

Amalia Gastaldelli (A)

Institute of Clinical Physiology, National Research Council (CNR), Pisa, Italy.

Paola Dongiovanni (P)

General Medicine and Metabolic Diseases, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy. Electronic address: paola.dongiovanni@policlinico.mi.it.

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Classifications MeSH