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The perforin pathway is not a single molecule but rather a cellular immune process involving the secretion of the pore-forming protein perforin by cytotoxic lymphocytes (natural killer cells and CD8+ T lymphocytes) in response to infected or transformed target cells[4][1][5][2]. Perforin is released at the immunological synapse between the effector and target cell, where it polymerizes in a calcium-dependent manner to form transmembrane pores in the target cell membrane[1][3][4][7]. These pores enable the entry of pro-apoptotic granzymes, such as granzyme B, which trigger apoptotic cell death of the target cell[5][6][4][2].\nDeficiencies or dysfunctions within the perforin pathway are linked to diseases such as familial hemophagocytic lymphohistiocytosis, immune deficiency, lymphoma, and certain autoimmune diseases[1][2][5]. While the pathway is a major component of immune surveillance against infection and malignancy, overactivation or genetic defects can result in immunopathology or ineffective immune responses[1][4][5].\nNote: "Perforin pathway" refers to a functional immune mechanism and not to a discrete molecular target; the canonical molecule in this pathway is perforin (gene name PRF1)[1][2][4]. Therefore, it is not considered a druggable target entity in the sense of a single receptor, enzyme, or ion channel, and detailed, structured drug-target data do not directly apply. For target-based information, see "perforin" or "PRF1".
Inhibition of perforin-mediated pore formation, Modulation of cytotoxic lymphocyte function
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