PIWI-interacting RNAs (piRNAs), a class of small noncoding RNAs, stabilize the genome at transcriptional and post-transcriptional levels. We identified and tested a number of tissue and cancer-type specific piRNAs as potential therapeutic candidates.

We profiled the expression of >23,000 piRNAs in the liver tissue and identified piRNAs that are under- or over-expressed in liver cancer relative to normal liver tissue (red dots in Fig.1A). We have demonstrated anti-cancer effects of down-regulated piR-37213-L01 both in vitro (cell proliferation, and colony formation) (Figure 1) and in-vivo (xenograft mouse models in Figure 2). The anti-cancer effect of piR-37213-L01 was highly specific for liver cancer and had no effect on other cancer types tested (breast, lung, glioma, prostate, etc.). Work involving testing piR-37213-L01 in PDX mouse models and uncovering the mechanism of action is under way.

Lead Innovator: Yong Zhu, Ph.D.

IP status: PCT/US17/19741 (50+ specific piRNA sequences for several cancer types).

References: Fu et al. 2015; Jacobs et al. 2016, Jacobs et al. 2018