Riassunto analitico
RNA interference (RNAi) has been suggested to represent a promising therapeutic approach in different disease settings. Primary effusion lymphoma (PEL) is a plasmablastic lymphoma consistently expressing B lymphocyte-induced maturation protein 1 (Blimp-1), a pivotal transcriptional regulator during terminal differentiation of B cells into plasma cells. We report, for the first time, that transient knockdown of BLIMP1 gene (also known as PR Domain Containing 1 with ZNF Domain, or PRDM1), using small interfering RNAs (siRNAs) delivered by cationic liposomes, was associated with efficient apoptosis induction and antiproliferative effect in PEL cell lines. We also showed that significantly prolonged survival in a PEL murine model was achieved by intraperitoneal treatment with such anti-BLIMP1 lipoplexes, while observing no vector-induced toxicity. This effective and safe RNAi strategy, based on liposomal siRNAs targeting a master transcription factor of post-germinal center B cells, may represent a potential treatment against plasmablastic lymphoma.
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