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Granulocyte-macrophage colony-stimulating factor gene therapy is a genetic medicine approach in which the GM-CSF gene is delivered, usually via plasmid DNA or viral/non-viral vectors, to host cells to induce local or systemic expression of GM-CSF protein. GM-CSF acts as an immunostimulatory cytokine, enhancing differentiation and activation of dendritic cells, macrophages, and other myeloid cells, resulting in enhanced immune response, especially anti-tumor immunity. This approach has been investigated as an adjuvant in cancer vaccines and to potentiate cellular immune responses against infection or cancer. Codon optimization and particular vector approaches are utilized to maximize GM-CSF protein production at the desired site, with the intended effect being increased antigen presentation and T cell-mediated immunity[3][1].
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