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Cancer Gene Ther. 2014 Feb;21(2):48-53. doi: 10.1038/cgt.2013.84. Epub 2014 Jan 24.

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

Author information

  • 1Department of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
  • 2Gradalis, Carrollton, TX, USA.
  • 31] Gradalis, Carrollton, TX, USA [2] Mary Crowley Cancer Research Center, Dallas, TX, USA.

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.

PMID:
24457987
[PubMed - indexed for MEDLINE]
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