Journal article
Targeting Nrf2 for the treatment of Duchenne Muscular Dystrophy
S Kourakis, CA Timpani, JB de Haan, N Gueven, D Fischer, E Rybalka
Redox Biology | Published : 2021
Open access
Abstract
Imbalances in redox homeostasis can result in oxidative stress, which is implicated in various pathological conditions including the fatal neuromuscular disease Duchenne Muscular Dystrophy (DMD). DMD is a complicated disease, with many druggable targets at the cellular and molecular level including calcium-mediated muscle degeneration; mitochondrial dysfunction; oxidative stress; inflammation; insufficient muscle regeneration and dysregulated protein and organelle maintenance. Previous investigative therapeutics tended to isolate and focus on just one of these targets and, consequently, therapeutic activity has been limited. Nuclear erythroid 2-related factor 2 (Nrf2) is a transcription fact..
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Funding Acknowledgements
This work was supported by funding from Victoria University and University Children's Hospital Basel.