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Restoring Neuromuscular Junction Integrity to Alleviate ALS Progression

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Technical Report,30 Sep 2018,29 Sep 2019

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University of Texas at Arlington Arlington United States

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ALS is a fatal neuromuscular disease. Failure of the respiratory muscle is a main cause of mortality in ALS patients. Defects in neuromuscular junctions NMJs and progressive NMJ loss occur at early stages, thus stabilizing and preserving NMJs represents a potential therapeutic strategy to slow ALS disease progression. This project is aim to explore a potential role of a muscle-derived protein MG53 in maintaining the NMJ and diaphragm muscle membrane integrity, and to establish the efficacy and safety for using recombinant human MG53 protein rhMG53 to treat ALS in the mouse model. Since MG53 is already present in human blood circulation under normal physiologic conditions, therapeutic approach with modulation of MG53 function or systemic administration of rhMG53 can potentially be a safe biologic reagent for treatment of ALS.

Subject Categories:

  • Medicine and Medical Research

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