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Amyotrophic lateral sclerosis (ALS)

Serine/threonine kinase 4 (STK4); superoxide dismutase 1 (SOD1)

Human tissue and mouse studies suggest STK4 inhibitors could help treat ALS. In spinal cord motor neurons from patients with sporadic ALS and mutant SOD1 transgenic mouse models for ALS, levels of activated STK4 were higher than those in neurons from healthy individuals and transgenic mice expressing wild-type SOD1. In the mutant SOD1 mouse model, homozygous Stk4 deletion slowed disease onset, increased motor neuron viability and neuromuscular function and decreased mortality compared with no deletion. Ongoing work includes identifying small molecule inhibitors of STK4.

SciBX 6(30); doi:10.1038/scibx.2013.797
Published online Aug. 8, 2013

Patent application filed; available for licensing

Lee, J.K. et al. Proc. Natl. Acad. Sci. USA; published online July 1, 2013;
Contact: Eui-Ju Choi, Korea University, Seoul, South Korea