Motor neurons are the cell type that is selectively lost in ALS. To develop tools to understand and monitor ALS disease progression, Mariah Hoye of Dr. Miller’s group identified factors that were specifically expressed in motor neurons only. These factors, called microRNAs (or miRNAs), may help researchers understand why motor neurons, and not other cell types, are selectively lost in ALS. One of the motor neuron enriched miRNAs was detected in spinal tap biofluid from an ALS rat model and its levels changed as disease progressed. These data suggest that miR-218 could be a clinically useful marker of disease status. Furthermore, Hoye found that rats treated with an ALS therapy had restored levels of the motor neuron marker, suggesting that miR-218 is a motor neuron specific and drug responsive marker for ALS rodents.
Hoye ML, Koval ED, Wegener AJ, Hyman TS, Yang C, O’Brien DR, Miller RL, Cole T, Schoch KM, Shen T, Kunikata T, Richard JP, Gutmann DH, Maragakis NJ, Kordasiewicz HB, Dougherty JD, Miller TM. MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models. J Neurosci. 2017 May 31;37(22):5574-5586. doi: 10.1523/JNEUROSCI.3582-16.2017. Epub 2017 Apr 17. PMID: 28416596; PMCID: PMC5452343.