Pax6 as a Therapeutic Target: Convergent Molecular Pathways in the Regulation of Aniridia, Neurogenesis, Neurodegeneration and Oncology

The transcription factor Pax6 acts as a molecular switch regulating various phases of the cell cycle during the early stages of embryonic neurogenesis and serves as a molecular mediator integrating multiple signaling pathways involved in the development of pathophysiological conditions. In this regard, Pax6 is considered an attractive molecular target for developing new drugs aimed at combating neurodegeneration, oncology, and aniridia. The aim of this review is to examine the range of studies describing various Pax6-dependent molecular pathways to identify similarities and differences in the signaling cascades activated by Pax6 in health and disease. These insights may provide a basis for developing new therapeutic strategies targeting Pax6 as a molecular regulator. This review summarizes the data indicating the leading regulatory role of Pax6 in the neuronal cell cycle in health and pathology. It is possible that Pax6 may act as a therapeutic target in certain pathophysiological conditions; however, the effectiveness of such a strategy will depend on the substrate chain in the signaling pathway, its branching, and the redundancy of mediators involved.