MicroRNAs as potential biomarkers for ALS.
Amyotrophic Lateral Sclerosis (ALS) is a devastating disease that progressively destroys motor neurons in the brain and the spinal cord, eventually causing paralysis and death. Currently, there are approximately 25,000 patients with ALS in the USA, with a median age of onset of 55 years. Approximately 5–10% of patients with ALS have a family history, and these patients most frequently inherit the disease in an autosomal dominant manner. Family-based linkage studies have led to the identification of several genes for familial ALS. However these findings only explain a small fraction of all ALS cases. The majority of ALS cases have no obvious family history and are referred to as sporadic ALS. At present, there is no effective therapy for the disease and patients usually die within 2-5 years after the onset of symptoms. Thus, there is an urgent need for biomarkers that could substantially aid early diagnosis of ALS and will help in designing decisive clinical trials of new drugs. The present technology provides specific microRNAs that can serve as potential biomarkers for ALS.
MicroRNAs (miRNAs) are endogenous small noncoding RNAs that negatively regulate gene expression in a posttranscriptional fashion and contribute to a wide variety of biological processes. miRNAs play important roles in the development of the central nervous system and their involvement in neurodegenerative diseases such as Parkinson's disease and Alzheimer’s disease has been recently established. The outlined technology describes specific miRNAs that are enriched in motor neurons and are significantly downregulated in mouse models of hereditary motor neuron disease (SOD1G93A and SMN1). These miRNAs may serve as putative biomarkers for motor neuron diseases such as ALS by measurement of their expression levels in cerebrospinal fluid samples collected from affected individuals.