Wilms tumor protein and Preferentially expressed antigen in melanoma (WT1 and PRAME)

WT1 (Wilms Tumor 1) and PRAME (Preferentially Expressed Antigen in Melanoma) are two distinct proteins that serve as critical leukemia-associated antigens (LAAs) frequently co-expressed in acute myeloid leukemia (AML) and other hematologic malignancies. WT1 is a zinc-finger transcription factor that regulates the expression of genes involved in cell growth, differentiation, and apoptosis; while it acts as a tumor suppressor in Wilms tumor, it is frequently overexpressed as an oncogene in leukemia and various solid tumors (1.1.1, 1.1.2). PRAME is a cancer-testis antigen that functions as a dominant repressor of retinoic acid receptor (RAR) signaling, thereby blocking cellular differentiation and promoting the survival and proliferation of malignant cells (1.2.1, 1.2.5). Both antigens are characterized by high expression in cancer cells with minimal expression in normal adult tissues, making them ideal targets for immunotherapy and sensitive biomarkers for monitoring minimal residual disease (MRD) (1.3.2, 1.4.2). Therapeutic strategies targeting these antigens include peptide vaccines (e.g., Galinpepimut-S), dendritic cell vaccines (e.g., FDC101), and engineered T-cell therapies such as TCR-T and CAR-T cells, which are often designed to target both antigens simultaneously to overcome tumor heterogeneity and prevent immune escape (1.4.1, 1.5.1).

Other names

Wilms tumor 1WT33AEWS-GUDNPHS4WAGRWIT-2CT130MAPEOIP4OIP-4Preferentially expressed antigen in melanomaWilms tumor protein

Immunotherapy (Vaccine, TCR-T, CAR-T, Bispecific T-cell engager) inducing T-cell mediated cytotoxicity against antigen-presenting tumor cells