psiRNA-SOX10

psiRNA-SOX10 is a gene therapy agent consisting of a plasmid vector-encoded small interfering RNA (siRNA), specifically designed to silence the Sry-related HMG-box gene 10 (SOX10), a transcription factor crucial for neural crest and melanoma cell function. Its primary mechanism is the targeted knockdown of SOX10 mRNA, leading to reduced expression of SOX10 protein, which in turn impairs melanoma cell proliferation, survival, melanogenesis, and tumor angiogenesis. In experimental mouse models, psiRNA-SOX10, often combined with a cationic lipid carrier (such as Lyovec), significantly decreased tumor mass and vascularization, particularly when used alongside a psiRNA targeting vascular endothelial growth factor (VEGF). The approach is experimental, developed for preclinical cancer gene therapy, particularly melanoma, aiming to enhance antiangiogenic and antitumor effects by disrupting tumor progression and microenvironment interactions[1].