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The mutant VHL peptide vaccine is an experimental cancer immunotherapy designed to treat patients with clear cell renal cell carcinoma (ccRCC) and other tumors associated with Von Hippel-Lindau (VHL) gene mutations. The VHL gene is a tumor suppressor that is inactivated or mutated in the vast majority of ccRCC cases, often resulting in the production of novel, tumor-specific neoantigens. This vaccine typically consists of synthetic peptides corresponding to the specific amino acid sequences created by these mutations, such as frameshift or missense mutations. When administered, usually in combination with an adjuvant like Montanide ISA-51 or cytokines like GM-CSF, the vaccine aims to prime and expand the population of cytotoxic T lymphocytes (CTLs) that can recognize these mutant epitopes presented on the surface of cancer cells via MHC class I molecules. By inducing a targeted cellular immune response, the vaccine seeks to selectively eliminate VHL-deficient tumor cells while sparing normal tissues.
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