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

Pax6 is a multifunctional transcription factor that orchestrates cell cycle progression at distinct stages of early embryonic neurogenesis and serves as a molecular mediator integrating multiple signaling pathways associated with pathological processes. Within this framework, Pax6 is regarded as an attractive molecular target for developing new drugs aimed at combating neurodegeneration, oncology, and aniridia. The present review aims to examine published studies describing various Pax6-dependent molecular pathways to identify common principles and condition-specific differences in Pax6-regulated cascades in health and disease. These insights may contribute to the conceptual foundation for developing new therapeutic strategies targeting Pax6 as a molecular regulator. This review summarizes the data demonstrating a central role of Pax6 in governing 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.