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Myc target protein 1 (MYCT1) is a membrane-associated protein identified as a direct downstream target of the oncogenic transcription factor MYC. MYCT1 plays an essential role in human hematopoietic stem and progenitor cells (HSPCs), where it regulates endocytosis, vesicle trafficking, and environmental sensing by localizing to endosomes via two transmembrane domains. Loss of MYCT1 disrupts HSC self-renewal, expansion, and engraftment, primarily through dysregulation of endocytosis and cell signaling. In cancer biology, MYCT1 exhibits complex roles, affecting cell proliferation, survival, migration, and tumor immune responses; its function can vary between acting as an oncogene or tumor suppressor depending on the context. MYCT1's relevance in disease includes links to hematologic malignancy and rare inherited neutropenia syndromes. Although not currently targeted by any approved drugs, the modulation of MYCT1 expression and activity has implications for the expansion of stem cell therapeutics and for understanding mechanisms of malignancy
null (No drugs are currently known to selectively target MYCT1)
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