Hoye ML, Regan MR, Jensen LA, Lake AM, Reddy LV, Vidensky S, Richard JP, Maragakis NJ, Rothstein JD, Dougherty JD, Miller TM. Motor neuron-derived microRNAs cause astrocyte dysfunction in amyotrophic lateral sclerosis. Brain. 2018 Sep 1;141(9):2561-2575. doi: 10.1093/brain/awy182. PMID: 30007309; PMCID: PMC6113638.
Mutations in superoxide dismutase 1 (SOD1) are responsible for 20% of familial ALS. Given the gain of toxic function in this dominantly inherited disease, lowering SOD1 mRNA and protein is predicted to provide therapeutic benefit. We have developed next-generation SOD1 ASOs that more potently reduce SOD1 mRNA and protein and extend survival in SOD1G93A mice. […]
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.