Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Neurology

Protein phosphatase 2 (PPP2CA; PP2A)

In vitro and mouse studies suggest inhibiting PPP2CA in the brain could help treat Opitz syndrome, an inherited neurological disorder. Midline 1 (MID1) is mutated in the X-linked form of Opitz syndrome and regulates the degradation of PPP2CA. In cultured primary mouse neurons, RNAi targeting Mid1 increased abnormal axon growth and levels of Ppp2ca compared with those seen in wild-type neurons, and knockout of Ppp2ca restored normal axon growth. In mice, genetic depletion of Mid1 disrupted normal axon growth, and a microRNA targeting Ppp2ca restored growth. Next steps include screening for inhibitors of PPP2CA.

SciBX 6(48); doi:10.1038/scibx.2013.1396
Published online Dec. 19, 2013

Findings unpatented; unavailable for licensing

Lu, T. et al. Proc. Natl. Acad. Sci. USA; published online Nov. 5, 2013;
doi:10.1073/pnas.1303687110
Contact: Zhi Xiong, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, China
e-mail:
xiongzhiqi@ion.ac.cn
Contact: Renchao Chen, same affiliation as above
e-mail:
rcchen@ion.ac.cn