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WT1-specific TCR gene-modified T cells (WT1 TCR-001) is an autologous adoptive cell therapy developed by University College London (UCL). The therapy consists of a patient's own T lymphocytes that have been retrovirally transduced to express a codon-optimized alpha and beta chain of a T-cell receptor (TCR) specific for the Wilms' Tumor 1 (WT1) antigen, restricted by HLA-A*0201. WT1 is an intracellular protein overexpressed in various hematological malignancies, including acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). By targeting peptide fragments of WT1 presented on MHC class I molecules, these engineered T cells can identify and eliminate leukemic cells that are otherwise inaccessible to conventional CAR-T therapies. The therapy is currently being evaluated in Phase I/II clinical trials for patients with AML, MDS, and tyrosine kinase inhibitor-resistant CML.
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