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Background: PIWI-interacting RNAs (piRNAs) are highly abundant small noncoding RNAs that partner with PIWI proteins to protect germline tissues from destabilizing transposon activity. While aberrant expression of PIWI proteins has been linked with poor outcomes for many cancer types, far less is known about the expression or function of piRNAs in a cancer context. Here we provide evidence supporting a novel role of piRNAs in gliomagenesis that may be leveraged for therapeutic benefit.

Methods: In order to characterize piRNA expression patterns in glioblastoma multiforme (GBM), we performed array-based piRNA expression profiling in seven pairs of human normal and tumor tissue specimens with validation by qPCR. In vitro (cellular proliferation, colony formation, cell cycle, apoptosis, transcriptional profiling) and in vivo (orthotopic xenograft mouse model) assays were performed to characterize the …