An expansion mutation in the C9orf72 gene causes ALS and frontotemporal dementia (FTD) through unknown means. The researchers have found that the expansion leads to the expression of proteins that would otherwise not exist within neurons. In this study, they will investigate whether and how these so-called c9- RAN proteins contribute to the disease. A better understanding of this could ultimately lead to development of therapeutics to reduce these proteins or their effects. The proteins may also serve as a biomarker for ALS development, and the team will investigate the utility of this approach.
|Effective start/end date||1/1/96 → 7/31/16|
- ALS Association: $240,000.00