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The ability of the WT1 protein to function as an antigenic target for immunotherapy has been examined in the murine model in vivo and the human system in vitro. In the murine system, the immunization with the 9-base peptide (a.a.235-243 CMTWNQMNL) or with WT1 DNA induced the proliferation of cytotoxic T lymphocytes (CTLs) specific for WT1.

In the human system in vitro, the stimulation of mononuclear cells by this peptide HLA2402 or HLA-A0201 increases the growth rate of CTLs specific for WT1. These results show that the WT1 protein can be used as an antigen for immunotherapy in cancer. Based on these results, this inmunotherapy has been introduced into clinical studies.