Restoring Neuromuscular Junction Integrity to Alleviate ALS Progression
Abstract
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.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 2020
- Accession Number
- AD1124969
Entities
People
- Jingsong Zhou
Organizations
- University of Texas at Arlington