Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

Mark E. Davis (), Jonathan E. Zuckerman, Chung Hang J. Choi, David Seligson, Anthony Tolcher, Christopher A. Alabi, Yun Yen, Jeremy D. Heidel and Antoni Ribas
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Mark E. Davis: Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA
Jonathan E. Zuckerman: Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA
Chung Hang J. Choi: Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA
David Seligson: Jonsson Comprehensive Cancer Center,
Anthony Tolcher: START - South Texas Accelerated Research Therapeutics, LLC, 4383 Medical Drive, 4th Floor, San Antonio, Texas 78229, USA
Christopher A. Alabi: Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA
Yun Yen: City of Hope Comprehensive Cancer Center, 1500 E. Duarte Road, Duarte, California 91010, USA
Jeremy D. Heidel: Calando Pharmaceuticals, 201 South Lake Avenue, Suite 703, Pasadena, California 91101, USA
Antoni Ribas: Jonsson Comprehensive Cancer Center,

Nature, 2010, vol. 464, issue 7291, 1067-1070

Abstract: Human RNAi therapy The ability to downregulate specific genes using systemically delivered short RNA molecules and the cellular mechanism known as RNA interference has been shown previously in mouse and non-human primate models. Davis et al. have now demonstrated for the first time in humans that a short interfering RNA (siRNA) molecule can be systemically delivered using nanoparticles to a solid tumour. The siRNA mediates directed cleavage of its target mRNA, thereby also reducing the protein level. This proof-of-principle study confirms the potential of this technology as a human therapeutic.

Date: 2010
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DOI: 10.1038/nature08956

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