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Short Communication

Targeting HPV16 DNA Using CRISPR/Cas Inhibits Anal Cancer Growth in Vivo

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Pages 475-482 | Received 23 Jan 2018, Accepted 19 Apr 2018, Published online: 12 Jun 2018
 

Abstract

Aim: The goal of this study was to determine if a single AAV vector, encoding Cas9 and guide RNAs specific for the HPV16 E6 and E7 genes, could inhibit the growth of an HPV16-induced tumor in vivo. Materials & methods: We grew HPV16+, patient-derived anal cancer explants in immunodeficient mice and then challenged these by injection of AAV-based vectors encoding Cas9 and control or HPV16-specific guide RNAs. Results & conclusion: We observed a significant and selective reduction in tumor growth when the HPV16 E6 and E7 genes were targeted using Cas9. These studies provide proof of principle for the hypothesis that CRISPR/Cas has the potential to be used to selectively treat HPV-induced tumors in humans.

Author’s contributions

DS Hsu, EM Kennedy and BR Cullen devised the initial idea for this study and planned the experiments. AVR Kornepati and W Glover performed the experiments. DS Hsu, AVR Kornepati and BR Cullen analyzed the results. DS Hsu and BR Cullen supervised the study and wrote and edited the paper.

Financial & competing interests disclosure

This work was supported by grant R21-CA198050 from the National Cancer Institute. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.