Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Parkinson's disease (PD)

Myocyte enhanced factor 2C (MEF2C)

In vitro studies suggest increasing MEF2C expression could help treat PD. In dopaminergic neurons derived from human induced pluripotent stem (iPS) cells expressing a-synuclein (SNCA) A53T, compared with cells expressing wild-type SNCA, mitochondrial-damaging pesticides linked to PD caused S-nitrosylation of MEF2C and decreased its expression. In dopaminergic neurons expressing mutant SNCA, pretreatment with an inhibitor of S-nitrosylation or isoxazole protected cells from toxin-induced apoptosis. Both compounds were identified in a screen for activators of neuronal MEF2 (D-MEF2) transcription. Next steps include testing whether the effects of pesticides are associated with other PD mutations.

SciBX 7(2); doi:10.1038/scibx.2014.63
Published online Jan. 16, 2014

Patents issued covering MEF2C and relevant neuroprotective agents; available for licensing

Ryan, S.D. et al. Cell; published online Nov. 27, 2013;
doi:10.1016/j.cell.2013.11.009
Contact: Stuart A. Lipton, Sanford-Burnham Medical Research Institute, La Jolla, Calif.
e-mail:
slipton@sbmri.org

Contact: Rajesh Ambasudhan, same affiliation as above
e-mail:
rajesh@sbmri.org

Contact: Rudolf Jaenisch, Whitehead Institute for Biomedical Research, Cambridge, Mass.
e-mail:
jaenisch@wi.mit.edu