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Vertically integrated translational studies of PDX1 as a therapeutic target for pancreatic cancer via a novel bifunctional RNAi platform

Cancer Gene Therapy volume 21, pages 48–53 (2014)Cite this article

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Abstract

RNA interference (RNAi) represents a powerful, new tool for scientific investigation as well as a promising new form of targeted gene therapy, with applications currently in clinical trials. Bifunctional short hairpin RNA (shRNA) are synthetic RNAi molecules, engineered to utilize multiple endogenous RNAi pathways to specifically silence target genes. Pancreatic and duodenal homeobox 1 (PDX1) is a key regulator of pancreatic development, β-cell differentiation, normal β-cell function and pancreatic cancer. Our aim is to review the process of identifying PDX1 as a specific, potential RNAi target in pancreatic cancer, as well as the underlying mechanisms and various forms of RNAi, with subsequent testing and development of PDX1-targeted bifunctional shRNA therapy.

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Acknowledgements

This research was supported by NCI Grant R01 CA95731, The Moss Foundation, Vivian Smith Foundation, MD Anderson Foundation and the H H Lee Research Program.

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Authors and Affiliations

  1. Department of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA

    J Wu, S Liu, J Yu, G Zhou, R Sanchez & F C Brunicardi

  2. Gradalis, Carrollton, TX, USA

    D Rao, C M Jay, P Kumar, N Templeton, N Senzer, P Maples & J Nemunaitis

  3. Mary Crowley Cancer Research Center, Dallas, TX, USA

    N Senzer & J Nemunaitis

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  1. J Wu
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Correspondence to F C Brunicardi.

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Competing interests

D Rao, CM Jay, P Kumar, N Senzer and N Templeton are employed by Gradalis. N Senzer, FC Brunicardi, D Rao and J Nemunaitis are shareholders in Gradalis.

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Wu, J., Liu, S., Yu, J. et al. Vertically integrated translational studies of PDX1 as a therapeutic target for pancreatic cancer via a novel bifunctional RNAi platform. Cancer Gene Ther 21, 48–53 (2014). https://doi.org/10.1038/cgt.2013.84

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