PAX6 (Paired box protein Pax-6) variants and mutations

PAX6 (also known as Paired box protein Pax-6) is a human protein-coding gene encoding a paired box protein Pax-6 protein. It orchestrates developmental gene programs in the eye, forebrain, and pancreatic endocrine system. Haploinsufficiency most classically causes aniridia and can also produce broader ocular, endocrine, and neurodevelopmental abnormalities. This analysis covers 938 PAX6 variants and mutations. Of these, 49% have computational variant effect predictions. Disease context includes isolated aniridia, Peters anomaly, and aniridia. Example PAX6 variants include M1I, M1L, and M1V.

Variant analysis overview

Variant and mutation evidence

Clinical, disease, and population context

Protein structure and variant hotspots

Data sources

Evidence in this analysis draws on EBI Proteins Variation, UniProt, gnomAD v4, EuropePMC, 3D Hotspot Analysis, Interaction Network Analysis, Protein Data Bank, AlphaFold DB, gnomAD constraint, Open Targets, ClinGen, MaveDB, LitVar.

Notable PAX6 variants

Examples include M1I, M1L, M1V, Q2*, Q2H, Q2R, N3S, S4I. Listed records include available protein-change notation, database identifiers, clinical classifications, computational predictions, population evidence, experimental measurements, and disease context.