Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Huntington's disease (HD)

NMDA receptor NR3A subtype (GRIN3A; NR3A); huntingtin (HTT)

Patient and mouse studies suggest inhibiting GRIN3A could help to prevent or delay HD progression. In patients with HD and in a mouse model of HD, synaptic expression of GRIN3A was increased compared with healthy individuals or mice. In the HD mouse model, Grin3a knockout decreased motor and cognitive dysfunction compared with no knockout and prevented striatal atrophy and synaptic disconnection. In a rat corticostriatal slice model, transfection of mutant HTT plus Grin3a led to decreased survival of spiny neurons compared with transfection of mutant HTT alone. Next steps include testing the protective effects of small interfering RNA−mediated knockdown of Grin3a in mouse models of HD after onset of disease pathology.

SciBX 6(31); doi:10.1038/scibx.2013.831
Published online Aug. 15, 2013

Not patented, licensing status not applicable

Marco, S. et al. Nat. Med.; published online July 14, 2013;
doi:10.1038/nm.3246
Contact: Isabel Pérez-Otaño, Center for Applied Medical Research at the University of Navarra, Pamplona, Spain
e-mail:

otano@unav.es