This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Alzheimer's disease (AD)

Legumain (LGMN; AEP)

Patient sample, tissue culture and in vitro studies suggest inhibiting AEP could help treat AD. In homogenates derived from patient frontal cortex samples, AEP activity was greater than that in samples from healthy controls. In rat hippocampal slices, acidosis activated AEP and led to microtubule-associated protein-t (MAPT; TAU; FTDP-17) hyperphosphorylation, a hallmark of AD. In a human neuronal cell line, small interfering RNA-mediated AEP knockdown resulted in decreased hyperphosphorylated TAU levels compared with no knockdown. Next steps include evaluating brain-specific knockdown of AEP in mouse models for AD and screening for small molecules that inhibit the protein.

SciBX 6(21); doi:10.1038/scibx.2013.522
Published online May 30, 2013

Unpatented;
licensing status not applicable

Basurto-Islas, G. et al. J. Biol. Chem.; published online May 2, 2013;
doi:10.1074/jbc.M112.446070
Contact: Khalid Iqbal, The Institute for Basic Research in Developmental Disabilities, Staten Island, N.Y.
e-mail:

khalid.iqbal.ibr@gmail.com