Adenylosuccinic acid: a novel inducer of the cytoprotectant Nrf2 with efficacy in Duchenne muscular dystrophy

Emma Rybalka; Craig A Goodman; Dean G Campelj; Alan Hayes; Cara A Timpani

doi:10.1080/03007995.2020.1865699

Adenylosuccinic acid: a novel inducer of the cytoprotectant Nrf2 with efficacy in Duchenne muscular dystrophy

Curr Med Res Opin. 2021 Mar;37(3):465-467. doi: 10.1080/03007995.2020.1865699. Epub 2021 Jan 29.

Authors

Emma Rybalka^{1

2}, Craig A Goodman^{2

3}, Dean G Campelj^{1

2}, Alan Hayes^{1

2

4}, Cara A Timpani^{1

2}

Affiliations

¹ Institute for Health and Sport, Victoria University, Melbourne, Australia.
² Australian Institute for Musculoskeletal Science, Victoria University, St Albans, Australia.
³ Center for Muscle Research, Department of Physiology, The University of Melbourne, Parkville, Australia.
⁴ Department of Medicine, Western Health, Melbourne Medical School, The University of Melbourne, Melbourne, Australia.

PMID: 33331789
DOI: 10.1080/03007995.2020.1865699

Abstract

Adenylosuccinic acid (ASA) modifies Duchenne muscular dystrophy (DMD) progression in dystrophic mdx mice and human DMD patients. Despite an established role for ASA in augmenting metabolism and cellular energy homeostasis, our previous data suggests an undiscovered ulterior mode of action capable of modifying DMD disease course. Here, we identify ASA as a novel inducer of nuclear factor erythroid 2-related factor-2 (Nrf2), master regulator of the antioxidant and cytoprotective response to cell stress.

Keywords: Duchenne muscular dystrophy; Nrf2; adenylosuccinic acid; therapeutics.

MeSH terms

Adenosine Monophosphate / analogs & derivatives
Animals
Disease Models, Animal
Humans
Mice
Mice, Inbred mdx
Muscle, Skeletal
Muscular Dystrophy, Duchenne* / drug therapy
Muscular Dystrophy, Duchenne* / genetics
NF-E2-Related Factor 2

Substances

NF-E2-Related Factor 2
adenylosuccinate
Adenosine Monophosphate