Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Huntington's disease (HD)

Potassium channel Kir4.1 (KCNJ10)

Mouse studies suggest increasing Kir4.1 expression could help treat HD. In mouse models of HD, striatal astrocytes were more depolarized and had lower Kir4.1 currents than those in wild-type mice. In a mouse model of HD, viral vector-mediated expression of Kir4.1 in astrocytes prolonged survival and improved motor properties; it also decreased striatal astrocyte depolarization and increased Kir4.1 currents compared with viral vector-mediated expression of a control protein. Next steps could include optimizing viral vectors for therapeutic delivery of Kir4.1 or designing compounds to augment channel activity.

SciBX 7(17); doi:10.1038/scibx.2014.497
Published online May 1, 2014

Unpatented; licensing status not applicable

Tong, X. et al. Nature Neurosci.; published online March 30, 2014;
doi:10.1038/nn.3691
Contact: Baljit S. Khakh, University of California, Los Angeles, Calif.
e-mail:

bkhakh@mednet.ucla.edu
Contact: Michael V. Sofroniew, same affiliation as above
e-mail:

sofroniew@mednet.ucla.edu