Growth differentiation factor 1 (GDF1)

Growth differentiation factor 1 (GDF1) is a secreted protein and a member of the transforming growth factor beta (TGF-β) superfamily that plays critical roles in embryonic development, especially in establishing left–right body asymmetry, cardiac morphogenesis, and neural tissue formation[4][5][6][2][3]. GDF1 is chiefly expressed in the embryonic nervous system and is a key modulator within the Nodal/TGF-β signaling pathway, where it cooperates with proteins like Nodal and Cripto to direct early lineage decisions and heart specification[4][5][6]. Genetic mutations of GDF1 are strongly linked to a spectrum of congenital heart defects (including heterotaxy, right atrial isomerism, and tetralogy of Fallot) and disorders of left-right patterning[7][6][5]. In adult tissues, GDF1 is largely silenced, but it can reactivate in disease states such as poorly differentiated hepatocellular carcinoma (HCC), where it contributes to tumor cell lineage plasticity, enhances metastasis, and alters response to immunotherapies[1]. Due to its dual genetic overlap, note that “CERS1” and some aliases (e.g., ceramide synthase 1, LAG1, LASS1) map to a distinct enzyme produced from the same bicistronic locus but are not functionally equivalent to GDF1 as a TGF-β ligand[4][5][6]; thus, the inclusion of these names refers to a gene context rather than the therapeutic target molecule itself. Clarification on 'is_incorrect': This target entry combines two unrelated proteins due to the peculiarity of its gene structure. "GDF1" refers to a TGF-β family ligand cytokine important in development, while "CERS1" (Ceramide synthase 1) is an enzyme produced from the same bicistronic gene, but with separate function and biology[4][5][6]. These should not be conflated; thus, this entry is flagged as "is_incorrect: true" for containing merged, unrelated targets. Only the TGF-β ligand (Growth differentiation factor 1) is typically considered a therapeutic target in the conventional sense.

Other names

Growth/differentiation factor 1GDF-1CERS1Ceramide synthase 1DORVDTGA3LAG1LASS1Longevity assurance gene 1 protein homolog 1Protein UOG-1UOG1