Wilms tumor protein 1 mRNA (WT1 mRNA) (WT1 mRNA)

Wilms tumor protein 1 (WT1) mRNA is the transcript of the WT1 gene, which encodes a zinc finger transcription factor crucial for urogenital development and often hijacked in oncogenesis (Scharnhorst et al., 2001, Gene). In the context of therapeutic development, WT1 mRNA is targeted through two primary modalities: as a template for cancer vaccines and as a substrate for antisense-mediated knockdown. As a vaccine component, WT1 mRNA is either directly injected or loaded into dendritic cells to induce a robust cytotoxic T-lymphocyte response against tumor cells overexpressing the WT1 protein (Van Driessche et al., 2005, Leukemia). Beyond its role as a therapeutic target, WT1 mRNA is a gold-standard biomarker for monitoring minimal residual disease (MRD) in acute myeloid leukemia (AML), where its levels correlate strongly with relapse risk (Cilloni & Saglio, 2012, Blood). The target is particularly attractive due to its high expression in various cancers—including AML, mesothelioma, and ovarian cancer—relative to limited expression in normal adult tissues. However, therapeutic strategies must account for potential off-target effects in the kidneys and hematopoietic system where WT1 plays a physiological role (National Cancer Institute, 2023).

Other names

Wilms tumor 1 mRNAWT1 transcriptWT1 messenger RNA

Induction of WT1-specific immune responses via translation of mRNA into protein (vaccines); sequence-specific binding and degradation of mRNA to inhibit protein synthesis (antisense).