This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Amyotrophic lateral sclerosis (ALS)

Fusion (involved in t(12;16) in malignant liposarcoma) (FUS; TLS)

Mouse studies suggest inhibiting FUS aggregation could help treat ALS. In mice, expression of an aggregation-prone FUS mutant resulted in motor neuron damage, motor neuron loss and eventual mouse death. In the mouse model, expression of the mutant protein led to pathogenic FUS inclusions throughout the nervous system and increased neuroinflammation compared with expression of wild-type FUS. Next steps could include identifying therapeutics that inhibit FUS aggregation.

SciBX 6(33); doi:10.1038/scibx.2013.899
Published online Aug. 29, 2013

Patent and licensing status unavailable

Shelkovnikova, T.A. et al. J. Biol. Chem.; published online July 18, 2013;
doi:10.1074/jbc.M113.492017
Contact: Natalia N. Ninkina, Cardiff University, Cardiff, U.K.
e-mail:

ninkinan@cf.ac.uk
Contact: Vladimir L. Buchman, same affiliation as above
e-mail:

buchmanvl@cf.ac.uk