This week in therapeutics

Indication

Target/marker/pathway

Summary

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Publication and contact information

Neurology

Down syndrome

Sorting nexin family member 27 (SNX27)

Patient sample and mouse studies suggest increasing SNX27 expression could help treat Down syndrome. In brain samples from patients with Down syndrome, SNX27 mRNA and protein levels were lower than those in healthy controls. In Snx27+/- mice, learning, memory and synaptic function were impaired. In a mouse model for Down syndrome, hippocampus injection of an adenovirus expressing human SNX27 rescued synaptic and cognitive deficits. Next steps include screening for compounds that activate SNX27 production.

SciBX 6(13); doi:10.1038/scibx.2013.316
Published online April 4, 2013

Findings unpatented; unavailable for licensing

Wang, X. et al. Nat. Med.;
published online March 24, 2013;
doi:10.1038/nm.3117
Contact: Huaxi Xu, Xiamen University, Xiamen, China
e-mail:
hxxu@xmu.edu.cn
Contact: Wanjin Hong, Sanford-Burnham Medical Research Institute,
La Jolla, Calif.
e-mail:
xuh@sanfordburnham.org