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Targeted gene silencing drugs are more than 500 times more effective with new delivery method

Date:
December 21, 2012
Source:
Mary Ann Liebert, Inc., Publishers
Summary:
Small interfering RNAs (siRNAs) are a potent new drug class that can silence a disease-causing gene, but delivering them to a target cell can be challenging. An innovative delivery approach dramatically increases the efficacy of an siRNA drug targeted to the liver and has made it possible to test the drug in non-human primates/

Small interfering RNAs (siRNAs) are a potent new drug class that can silence a disease-causing gene, but delivering them to a target cell can be challenging. An innovative delivery approach that dramatically increases the efficacy of an siRNA drug targeted to the liver and has made it possible to test the drug in non-human primates is described in an article in Nucleic Acid Therapeutics.

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In a new article Wong and colleagues from Arrowhead Madison Inc. (Madison, WI) present a novel strategy to overcome the difficulty in delivering high levels of a gene knockdown siRNA drug to liver cells. While the cholesterol-conjugated siRNA drug is taken up preferentially by the liver, it is encapsulated in membrane-bound globules called endosomes and cannot reach the cells' DNA to exert its gene silencing effect. The researchers co-injected a polymer with the drug that also targets the liver and, once inside liver cells, breaks open the endosomes, releasing the siRNA drug.

"The promise of siRNAs is as strong as ever and is becoming even more so with progress in delivering these molecules to the right place at the right time," says Executive Editor Fintan Steele, PhD, SomaLogic, Inc., Boulder, CO. "The work by Wong and colleagues is another important step towards realizing this promise."


Story Source:

The above story is based on materials provided by Mary Ann Liebert, Inc., Publishers. Note: Materials may be edited for content and length.


Journal Reference:

  1. So C. Wong, Jason J. Klein, Holly L. Hamilton, Qili Chu, Christina L. Frey, Vladimir S. Trubetskoy, Julia Hegge, Darren Wakefield, David B. Rozema, and David L. Lewis. Co-Injection of a Targeted, Reversibly Masked Endosomolytic Polymer Dramatically Improves the Efficacy of Cholesterol-Conjugated Small Interfering RNAs In Vivo. Nucleic Acid Therapeutics, December 2012, 22(6): 380-390 DOI: 10.1089/nat.2012.0389

Cite This Page:

Mary Ann Liebert, Inc., Publishers. "Targeted gene silencing drugs are more than 500 times more effective with new delivery method." ScienceDaily. ScienceDaily, 21 December 2012. <www.sciencedaily.com/releases/2012/12/121221123418.htm>.
Mary Ann Liebert, Inc., Publishers. (2012, December 21). Targeted gene silencing drugs are more than 500 times more effective with new delivery method. ScienceDaily. Retrieved March 4, 2015 from www.sciencedaily.com/releases/2012/12/121221123418.htm
Mary Ann Liebert, Inc., Publishers. "Targeted gene silencing drugs are more than 500 times more effective with new delivery method." ScienceDaily. www.sciencedaily.com/releases/2012/12/121221123418.htm (accessed March 4, 2015).

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