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P300 shRNA is a gene-silencing therapeutic candidate designed to knock down the expression of the transcriptional co-activator P300 (EP300). P300 is a histone acetyltransferase that regulates gene expression through histone H3 lysine 27 acetylation (H3K27ac) and is implicated in the progression of multiple myeloma (MM) and the formation of extra-medullary disease (EMD). By utilizing a lentiviral delivery system to introduce short hairpin RNA (shRNA), this approach aims to inhibit MM cell proliferation, induce cell cycle arrest in the G0/G1 phase, and reduce the invasive and migratory capacity of malignant cells. Preclinical studies conducted at Massachusetts General Hospital and Harvard Medical School have demonstrated that P300 silencing reduces tumor homing to the liver and kidneys in mouse models, suggesting its potential as a therapeutic target for relapsed or disseminated myeloma.
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