C9RANT is a newly discovered protein-like chain that holds great potential promise as a benchmark for measuring disease activity and therapeutic response for patients with the devastating conditions of amyotrophic lateral sclerosis (ALS) and dementia. Mutations within a non-coding region of the C9ORF72gene are responsible for 20-40% of cases of familial ALS1. This class of mutations generates repetitive, high molecular weight RNA chains which are insoluble and aggregate within the brain as C9RANT accumulations.
