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PELP1 siRNA (also known as PELP1-siRNA-DOPC) is a preclinical-stage RNA interference (RNAi) therapeutic consisting of a small interfering RNA sequence targeting the Proline-, Glutamate- and Leucine-rich Protein 1 (PELP1) gene. PELP1 is a nuclear receptor coregulator and proto-oncogene that is overexpressed in several malignancies, including glioblastoma, breast, ovarian, and gastric cancers, where it facilitates tumor progression and metastasis through the modulation of hormonal signaling and the β-catenin pathway. To enable systemic delivery, the siRNA is typically encapsulated in neutral nanoliposomes composed of 1,2-dioleoyl-sn-glycero-3-phosphatidylcholine (DOPC). Research conducted primarily at the University of Texas Health Science Center at San Antonio has demonstrated that silencing PELP1 with this therapeutic approach significantly inhibits cell proliferation, migration, and in vivo tumor growth in various xenograft models.
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