A coordinated multiorgan metabolic response contributes to human mitochondrial myopathy

Abstract Mitochondrial diseases are a heterogeneous group of monogenic disorders that result from impaired oxidative phosphorylation (OXPHOS). As neuromuscular tissues are highly energy‐dependent, mitochondrial diseases often affect skeletal muscle. Although genetic and bioenergetic causes of OXPHOS...

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Main Authors: Nneka Southwell, Guido Primiano, Viraj Nadkarni, Nabeel Attarwala, Emelie Beattie, Dawson Miller, Sumaitaah Alam, Irene Liparulo, Yevgeniya I Shurubor, Maria Lucia Valentino, Valerio Carelli, Serenella Servidei, Steven S Gross, Giovanni Manfredi, Qiuying Chen, Marilena D'Aurelio
Format: Article
Language:English
Published: Springer Nature 2023-05-01
Series:EMBO Molecular Medicine
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Online Access:https://doi.org/10.15252/emmm.202216951
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author Nneka Southwell
Guido Primiano
Viraj Nadkarni
Nabeel Attarwala
Emelie Beattie
Dawson Miller
Sumaitaah Alam
Irene Liparulo
Yevgeniya I Shurubor
Maria Lucia Valentino
Valerio Carelli
Serenella Servidei
Steven S Gross
Giovanni Manfredi
Qiuying Chen
Marilena D'Aurelio
author_facet Nneka Southwell
Guido Primiano
Viraj Nadkarni
Nabeel Attarwala
Emelie Beattie
Dawson Miller
Sumaitaah Alam
Irene Liparulo
Yevgeniya I Shurubor
Maria Lucia Valentino
Valerio Carelli
Serenella Servidei
Steven S Gross
Giovanni Manfredi
Qiuying Chen
Marilena D'Aurelio
author_sort Nneka Southwell
collection DOAJ
description Abstract Mitochondrial diseases are a heterogeneous group of monogenic disorders that result from impaired oxidative phosphorylation (OXPHOS). As neuromuscular tissues are highly energy‐dependent, mitochondrial diseases often affect skeletal muscle. Although genetic and bioenergetic causes of OXPHOS impairment in human mitochondrial myopathies are well established, there is a limited understanding of metabolic drivers of muscle degeneration. This knowledge gap contributes to the lack of effective treatments for these disorders. Here, we discovered fundamental muscle metabolic remodeling mechanisms shared by mitochondrial disease patients and a mouse model of mitochondrial myopathy. This metabolic remodeling is triggered by a starvation‐like response that evokes accelerated oxidation of amino acids through a truncated Krebs cycle. While initially adaptive, this response evolves in an integrated multiorgan catabolic signaling, lipid store mobilization, and intramuscular lipid accumulation. We show that this multiorgan feed‐forward metabolic response involves leptin and glucocorticoid signaling. This study elucidates systemic metabolic dyshomeostasis mechanisms that underlie human mitochondrial myopathies and identifies potential new targets for metabolic intervention.
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spelling doaj-art-3ac8c9834e7f48439c744d6d9391fd6b2025-08-20T03:43:26ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842023-05-0115712610.15252/emmm.202216951A coordinated multiorgan metabolic response contributes to human mitochondrial myopathyNneka Southwell0Guido Primiano1Viraj Nadkarni2Nabeel Attarwala3Emelie Beattie4Dawson Miller5Sumaitaah Alam6Irene Liparulo7Yevgeniya I Shurubor8Maria Lucia Valentino9Valerio Carelli10Serenella Servidei11Steven S Gross12Giovanni Manfredi13Qiuying Chen14Marilena D'Aurelio15Brain and Mind Research Institute, Weill Cornell MedicineFondazione Policlinico Universitario Agostino Gemelli IRCCSBrain and Mind Research Institute, Weill Cornell MedicineDepartment of Pharmacology, Weill Cornell MedicineBrain and Mind Research Institute, Weill Cornell MedicineDepartment of Pharmacology, Weill Cornell MedicineDepartment of Pharmacology, Weill Cornell MedicineBrain and Mind Research Institute, Weill Cornell MedicineBrain and Mind Research Institute, Weill Cornell MedicineIRCCS, Institute of Neurological Sciences of Bologna, Bellaria HospitalIRCCS, Institute of Neurological Sciences of Bologna, Bellaria HospitalFondazione Policlinico Universitario Agostino Gemelli IRCCSDepartment of Pharmacology, Weill Cornell MedicineBrain and Mind Research Institute, Weill Cornell MedicineDepartment of Pharmacology, Weill Cornell MedicineBrain and Mind Research Institute, Weill Cornell MedicineAbstract Mitochondrial diseases are a heterogeneous group of monogenic disorders that result from impaired oxidative phosphorylation (OXPHOS). As neuromuscular tissues are highly energy‐dependent, mitochondrial diseases often affect skeletal muscle. Although genetic and bioenergetic causes of OXPHOS impairment in human mitochondrial myopathies are well established, there is a limited understanding of metabolic drivers of muscle degeneration. This knowledge gap contributes to the lack of effective treatments for these disorders. Here, we discovered fundamental muscle metabolic remodeling mechanisms shared by mitochondrial disease patients and a mouse model of mitochondrial myopathy. This metabolic remodeling is triggered by a starvation‐like response that evokes accelerated oxidation of amino acids through a truncated Krebs cycle. While initially adaptive, this response evolves in an integrated multiorgan catabolic signaling, lipid store mobilization, and intramuscular lipid accumulation. We show that this multiorgan feed‐forward metabolic response involves leptin and glucocorticoid signaling. This study elucidates systemic metabolic dyshomeostasis mechanisms that underlie human mitochondrial myopathies and identifies potential new targets for metabolic intervention.https://doi.org/10.15252/emmm.202216951amino acid metabolismglucocorticoidsleptinmitochondrial myopathymuscle wasting
spellingShingle Nneka Southwell
Guido Primiano
Viraj Nadkarni
Nabeel Attarwala
Emelie Beattie
Dawson Miller
Sumaitaah Alam
Irene Liparulo
Yevgeniya I Shurubor
Maria Lucia Valentino
Valerio Carelli
Serenella Servidei
Steven S Gross
Giovanni Manfredi
Qiuying Chen
Marilena D'Aurelio
A coordinated multiorgan metabolic response contributes to human mitochondrial myopathy
EMBO Molecular Medicine
amino acid metabolism
glucocorticoids
leptin
mitochondrial myopathy
muscle wasting
title A coordinated multiorgan metabolic response contributes to human mitochondrial myopathy
title_full A coordinated multiorgan metabolic response contributes to human mitochondrial myopathy
title_fullStr A coordinated multiorgan metabolic response contributes to human mitochondrial myopathy
title_full_unstemmed A coordinated multiorgan metabolic response contributes to human mitochondrial myopathy
title_short A coordinated multiorgan metabolic response contributes to human mitochondrial myopathy
title_sort coordinated multiorgan metabolic response contributes to human mitochondrial myopathy
topic amino acid metabolism
glucocorticoids
leptin
mitochondrial myopathy
muscle wasting
url https://doi.org/10.15252/emmm.202216951
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