A4GALT encodes α1,4-galactosyltransferase, a Golgi membrane-resident enzyme that catalyzes the synthesis of globotriaosylceramide (Gb3) glycosphingolipids and P1 antigens on glycoproteins 1. The enzyme exhibits dual acceptor specificity, synthesizing both glycosphingolipids and modifying glycoproteins through homodimerization and heterodimerization with other galactosyltransferases 1. A4GALT expression levels determine P1/P2 blood group phenotypes, with single nucleotide polymorphisms in the 5' untranslated region affecting transcript levels and enzyme activity 23. The enzyme's products serve as cellular receptors for Shiga toxins, facilitating toxin entry and subsequent cellular damage. In Fabry disease, where α-galactosidase A is deficient, A4GALT contributes to pathological Gb3 accumulation. CRISPR/Cas9-mediated A4GALT suppression effectively rescues Fabry disease phenotypes in kidney organoids and endothelial cells by reducing Gb3 deposition and restoring cellular function 45. Additionally, siRNA-mediated A4GALT knockdown shows therapeutic promise for Fabry disease treatment 6. These findings establish A4GALT as both a critical determinant of blood group antigens and a potential therapeutic target for glycosphingolipid storage disorders.