Journal ArticleDOI
What skeletal muscle has to say in amyotrophic lateral sclerosis: Implications for therapy.
Raquel Manzano,Janne M. Toivonen,Laura Moreno-Martínez,Miriam de la Torre,Leticia Moreno-García,Tresa López-Royo,Nora Molina,Pilar Zaragoza,Ana C. Calvo,Rosario Osta +9 more
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TLDR
In this paper, a review aims to compile scientific evidence that demonstrates the role of skeletal muscle in ALS pathology and serves as reference for development of novel therapeutic strategies targeting this tissue to delay disease onset and progression.Abstract:
Amyotrophic lateral sclerosis (ALS) is an adult onset disorder characterized by progressive neuromuscular junction (NMJ) dismantling and degeneration of motor neurons leading to atrophy and paralysis of voluntary muscles responsible for motion and breathing. Except for a minority of patients harbouring genetic mutations, the origin of most ALS cases remains elusive. Peripheral tissues, and particularly skeletal muscle, have lately demonstrated an active contribution to disease pathology attracting a growing interest for these tissues as therapeutic targets in ALS. In this sense, molecular mechanisms essential for cell and tissue homeostasis have been shown to be deregulated in the disease. These include muscle metabolism and mitochondrial activity, RNA processing, tissue-resident stem cell function responsible for muscle regeneration, and proteostasis that regulates muscle mass in adulthood. This review aims to compile scientific evidence that demonstrates the role of skeletal muscle in ALS pathology and serves as reference for development of novel therapeutic strategies targeting this tissue to delay disease onset and progression. LINKED ARTICLES: This article is part of a themed issue on Neurochemistry in Japan. To view the other articles in this section visit http://onlinelibrary.wiley.com/doi/10.1111/bph.v178.6/issuetoc.read more
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C9orf72 and RAB7L1 regulate vesicle trafficking in amyotrophic lateral sclerosis and frontotemporal dementia (vol 140, pg 887, 2017)
Yoshitsugu Aoki,Raquel Manzano,Yi Lee,Ruxandra Dafinca,M Aoki,Douglas Agl.,Miguel A. Varela,Chaitra Sathyaprakash,Jakub Scaber,Paola Barbagallo,Pieter Vader,Imre Mäger,Kariem Ezzat,Martin R Turner,Naoki Ito,Samanta Gasco,Norihiko Ohbayashi,S El Andaloussi,Shin'ichi Takeda,Mitsunori Fukuda,Kevin Talbot,Wood Mja. +21 more
TL;DR: In this article, the authors reported a novel disease mechanism arising due to the interaction of C9ORF72 with the RAB7L1 GTPase to regulate vesicle trafficking.
Journal ArticleDOI
The Skeletal Muscle Emerges as a New Disease Target in Amyotrophic Lateral Sclerosis
Oihane Pikatza-Menoio,Amaia Elicegui,Xabier Bengoetxea,Neia Naldaiz-Gastesi,Adolfo López de Munain,Gorka Gerenu,Gorka Gerenu,Francisco Javier Gil-Bea,Sonia Alonso-Martín +8 more
TL;DR: In this article, the role of skeletal muscle in ALS through backward signaling processes was investigated, highlighting its contribution to the neurodegeneration through backward-signaling processes as a newly uncovered mechanism for a peripheral etiopathogenesis of the disease.
Journal ArticleDOI
Walking down Skeletal Muscle Lane: From Inflammasome to Disease
Nicolas Dubuisson,Nicolas Dubuisson,Romain Versele,María América Davis López de Carrizosa,María América Davis López de Carrizosa,Camille M. Selvais,Sonia Brichard,Michel Abou-Samra +7 more
TL;DR: In this article, the authors present the current knowledge regarding the function of NLRP3 in diseased skeletal muscle, and discuss the potential therapeutic options targeting the inflammasome in muscle disorders.
Journal ArticleDOI
ALS mutations in both human skeletal muscle and motoneurons differentially affects neuromuscular junction integrity and function.
TL;DR: In this article , human skeletal muscle (hSKM) and human motoneurons (hMN) generated from induced pluripotent stem cells of healthy individuals (WT) and ALS patients with two different SOD1 mutations were integrated into functional NMJ systems to investigate and compare the pathological contribution of the hSKM and hMN to ALS NMJ disruption.
Journal ArticleDOI
Therapeutic Targets in Amyotrophic Lateral Sclerosis: Focus on Ion Channels and Skeletal Muscle
TL;DR: This review summarizes the already tested drugs aimed at restoring muscle-nerve cross-talk and on new treatment options targeting this tissue to help reverse the damage in this devastating pathology.
References
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Mitochondrial Reactive Oxygen Species (ROS) and ROS-Induced ROS Release
TL;DR: The mechanism of mitochondrial RIRR highlights the central role of mitochondria-formed ROS, and all of the known ROS-producing sites and their relevance to the mitochondrial ROS production in vivo are discussed.
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Onset and Progression in Inherited ALS Determined by Motor Neurons and Microglia
Séverine Boillée,Koji Yamanaka,Christian S. Lobsiger,Neal G. Copeland,Nancy A. Jenkins,George Kassiotis,George Kollias,Don W. Cleveland +7 more
TL;DR: Onset and progression of amyotrophic lateral sclerosis represent distinct disease phases defined by mutant action within different cell types to generate non–cell-autonomous killing of motor neurons; these findings validate therapies, including cell replacement, targeted to the non-neuronal cells.
Journal ArticleDOI
ALS: a disease of motor neurons and their nonneuronal neighbors.
TL;DR: In this paper, a mutant superoxide dismutase (SOD1) was found to induce non-cell-autonomous motor neuron killing by an unknown gain of toxicity.
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Amyotrophic lateral sclerosis is a distal axonopathy: evidence in mice and man.
Lindsey R. Fischer,Deborah G. Culver,Philip Tennant,Albert A. Davis,Minsheng Wang,Amilcar A. Castellano-Sanchez,Jaffar M. Khan,Meraida Polak,Jonathan D. Glass +8 more
TL;DR: It is concluded that in this widely studied animal model of human ALS, and in this single human case, motor neuron pathology begins at the distal axon and proceeds in a "dying back" pattern.
Journal ArticleDOI
Wild-type nonneuronal cells extend survival of SOD1 mutant motor neurons in ALS mice.
A. M. Clement,Minh Dang Nguyen,E. A. Roberts,E. A. Roberts,M. L. Garcia,M. L. Garcia,Séverine Boillée,Séverine Boillée,M. Rule,Andrew P. McMahon,Wilder Doucette,D. Siwek,Robert J. Ferrante,Robert H. Brown,Jean-Pierre Julien,Lawrence S.B. Goldstein,Lawrence S.B. Goldstein,Don W. Cleveland,Don W. Cleveland +18 more
TL;DR: Nonneuronal cells that do not express mutant SOD1 delay degeneration and significantly extend survival of mutant-expressing motor neurons.