Compositions and methods of targeting the pax6 signaling pathway to reduce formation of amyloid beta plaques and neurofibrillary tangles

Abstract

Compositions and method of reducing Tau phosphorylation in neurons of a subject in need thereof are provided. In some embodiments, the subject has a proteinopathy, amyloidosis, or a tauopathy. Thus, compositions and methods of treating a proteinopathy, amyloidosis, or a tauopathy are also provided. Also disclosed are compositions and methods of increasing learning and/or memory in a subject with a tauopathy. The methods typically include administering the subject an effective amount of a direct or indirect inhibitor of Pax6 (Pax6 inhibitor). The Pax6 inhibitor can be, for example, a small molecule or a functional nucleic acid. In some embodiments, the small molecule is palbociclib, apigenin, flavopiridol, abemaciclib, ribociclib, or ICCB280, or a derivative, stereoisomer, or pharmaceutically acceptable salt thereof. In some embodiments, the functional nucleic acid including but not limited to siRNA, shRNA, or miRNA, or a nucleic acid expression construct encoding an siRNA, shRNA, or miRNA.

Dependencies are inferred from the claim text.