Therapeutics Targeting Skeletal Muscle in Amyotrophic Lateral Sclerosis

Author:

Gao Jinghui1,Sterling Elijah1,Hankin Rachel1ORCID,Sikal Aria1,Yao Yao1ORCID

Affiliation:

1. Regenerative Bioscience Center, Department of Animal and Dairy Science, College of Agricultural and Environmental Science, University of Georgia, Athens, GA 30602, USA

Abstract

Amyotrophic lateral sclerosis (ALS) is a complex neuromuscular disease characterized by progressive motor neuron degeneration, neuromuscular junction dismantling, and muscle wasting. The pathological and therapeutic studies of ALS have long been neurocentric. However, recent insights have highlighted the significance of peripheral tissue, particularly skeletal muscle, in disease pathology and treatment. This is evidenced by restricted ALS-like muscle atrophy, which can retrogradely induce neuromuscular junction and motor neuron degeneration. Moreover, therapeutics targeting skeletal muscles can effectively decelerate disease progression by modulating muscle satellite cells for muscle repair, suppressing inflammation, and promoting the recovery or regeneration of the neuromuscular junction. This review summarizes and discusses therapeutic strategies targeting skeletal muscles for ALS treatment. It aims to provide a comprehensive reference for the development of novel therapeutics targeting skeletal muscles, potentially ameliorating the progression of ALS.

Funder

Department of Defense Congressionally Directed Medical Research Programs Amyotrophic Lateral Sclerosis Research Program Therapeutic Idea Award

University of Georgia

Publisher

MDPI AG

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