Description:
- Novel microprotein therapeutic that restores mitochondrial function to address neurodegeneration
- Reduces neuroinflammation and improves brain metabolism for broad neuroprotective effects
- Demonstrated efficacy in in vitro and in vivo models of neurodegenerative disease
Abstract
USC researchers have identified SHMOOSE, a novel mitochondrial microprotein implicated in Alzheimer’s disease and other neurodegenerative disorders, as a promising therapeutic target. Synthetic SHMOOSE analogues were developed to restore mitochondrial function, reduce neuroinflammation, improve brain metabolism, and preserve cognitive function. Certain SHMOOSE variants are associated with increased disease risk, supporting a genetically informed therapeutic approach. Demonstrated efficacy in in vitro and in vivo models highlights the potential of these microprotein-based therapeutics to slow disease progression and provide a disease-modifying treatment strategy for Alzheimer’s disease and other neurodegenerative disorders.
Benefit
- Novel Therapeutic Class: Microproteins are a new class of therapeutics, enabling potential first-to-market opportunities.
- Unique Target: SHMOOSE targets mitochondrial dysfunction—a critical pathway in neurodegeneration.
- Restores Brain Health: Improves mitochondrial function and reduces inflammation for broad neuroprotection.
- Genetically Informed: Certain SHMOOSE variants are linked to higher disease risk, enabling a precision medicine approach.
- Broad Disease Application: Mitochondrial dysfunction is common across neurodegenerative diseases, offering cross-indication potential.
Market Application
- Alzheimer’s disease
- Parkinson’s disease and other neurodegenerative disorders associated with mitochondrial dysfunction
- Mitochondria-targeted therapeutics and precision medicine for CNS diseases
Publications
https://pmc.ncbi.nlm.nih.gov/articles/PMC10027624
Other
- In vivo and in vitro studies completed, which showed SHMOOSE’s effects on improving neurodegenerative pathology.