Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Huntington's disease (HD)

Huntingtin (HTT); translocase of inner mitochondrial membrane 23 homolog (TIMM23; TIM23)

Mouse and cell culture studies suggest increasing TIM23 activity or mitochondrial protein import could help treat HD. In cell culture, mutant Htt interacted with Tim23 and inhibited mitochondrial protein import, resulting in neuronal cell death. In mutant Htt-expressing primary mouse neurons, lentivirus-mediated overexpression of the Tim23 complex rescued the mitochondrial protein import defect and decreased cell death compared with overexpression of a control gene. Next steps could include screening for pharmacological compounds that increase TIM23 activity and mitochondrial protein transport.

SciBX 7(24); doi:10.1038/scibx.2014.718
Published online June 19, 2014

Patent and licensing status unavailable

Yano, H. et al. Nat. Neurosci.;
published online May 18, 2014;
doi:10.1038/nn.3721
Contact: Robert M. Friedlander, University of Pittsburgh, Pittsburgh, Pa.
e-mail:
friedlanderr@upmc.edu