Jiou Wang, MD, PhD
Molecular mechanisms of pathogenesis in ALS
Our laboratory seeks to use the small, soil-living, transparent roundworm – Caenorhabditis elegans – as a model organism to clarify how disease genes cause ALS in humans. Like the fruitfly, C. elegans is used by researchers to investigate how both healthy and mutated genes affect function. With its fast growth rate and the amount of knowledge we already hold about its genome, the tiny animal – it's about the width of a pencil lead – provides a unique system to study aging related diseases.
Recently, we genetically engineered C. elegans to model one specific form of inherited ALS (that with mutant SOD1 genes). Using the C. elegans model, we were able to conduct unbiased, large-scale screens to search for uncommon genes that might slightly raise the risk of having the disease. Now we aim to establish new C. elegans models of ALS linked to those mutations in patients. We hope that our studies will reveal important disease pathways in humans, and thus provide potential drug targets.
Note: Funding for this project was made possible by the Muscular Dystrophy Association.