Disease Mechanisms and Therapeutics for ALS: From Animal Models to Humans

Dear Colleagues,

Amyotrophic lateral sclerosis (ALS) is an incurable neuromuscular disease characterized by the selective death of motor neurons, resulting in progressive muscle atrophy. There is an emergent need to develop therapeutic strategies because of the modest effects of Riluzole and Edaravone, approved drugs for use in ALS, on the disease process. Success of developing animal models based on genetic discovery provides an understanding of molecular mechanisms underlying the pathogenesis of ALS as well as an opportunity to identify a potential candidate for treatment. Much effort has been devoted to discovering responsible genes for ALS such as SOD1, TARDBP, FUS, and C9ORF72. Nevertheless, our understanding of the disease process and the development of therapeutics for ALS can be still space for improvement.

This Special Issue welcomes original research with focus on a proof-concept study for a disease mechanism of ALS as well as a preclinical test using either mammalian or non-mammalian models carrying any responsible genes for ALS. Review articles that summarizes the ALS pathogenesis and/or preclinical outcomes based on animal models of ALS are also welcome. This Special Issue will advance our understanding of disease pathogenesis and develop therapeutic strategies for ALS with your research.

Dr. Eiichi Tokuda
Guest Editor

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