Navigation Links
Previous claims of siRNA therapeutic effects called into question by report in human gene therapy

New Rochelle, NY, September 2, 2008The many recent reports documenting the therapeutic efficacy of short interfering RNAs (siRNAs) in animal models of human disease may actually be describing non-specific therapeutic effects related to the ability of siRNA to activate an immune response, according to a paper in the September 2008 issue (Volume 19, Number 9) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. The paper, which was published "instant online," is available free online at

Marjorie Robbins, Adam Judge, Ellen Ambegia, Catherine Choi, Ed Yaworski, Lorne Palmer, Kevin McClintock, and Ian MacLachaln of Protiva Biotherapeutics (Burnaby, BC Canada), in a paper entitled, "Misinterpreting the Therapeutic Effects of siRNA Caused by Immune Stimulation," emphasize the need for researchers to design siRNA studies that incorporate suitable controls to differentiate the disease-modulating effect of an siRNA from its ability to stimulate an innate immune response.

siRNAs have been highly touted for their ability to target very specifically and selectively the disease-causing factors in a range of disorders, from viral infections to tumors and inflammatory and immunologic processes. However, siRNA also has the potential to activate innate immunity and the production of interferons, which can in turn bring about therapeutic effects in a range of disease models.

The authors of this paper contend that, "surprisingly few of the reported studies have adequately tested, or controlled, for the potential effects of siRNA-mediated immune stimulation."

In the current study, use of a commonly used control siRNA sequence called GFP siRNA, which has only a minimal capacity to activate the immune system, clearly showed the striking difference between the immunostimulatory potential of GFP siRNA and of some other siRNAs. Using a mouse model of influenza, the authors demonstrated that the anti-viral activity of siRNA is mainly due to non-specific stimulation of the immune system rather than to a targeted attack on the disease-causing virus.

"siRNA holds tremendous potential as a research tool, however its clinical development is still in its infancy. The study by Robbins et al. points out a very important issue regarding non-specific effects that should be considered when designing and evaluating siRNA strategies," says James M. Wilson, MD, PhD, Editor-in-Chief, and Head of the Gene Therapy Program, Division of Medical Genetics, University of Pennsylvania School of Medicine, in Philadelphia.


Contact: Vicki Cohn
Mary Ann Liebert, Inc./Genetic Engineering News

Related biology news :

1. Setting the record right: species diversity less dramatic than previously believed
2. Species have come and gone at different rates than previously believed
3. Galiximab in combination with rituximab in patients with previously untreated follicular lymphoma
4. Bacterial infections in premature babies more common than previously realized
5. Pathogens use previously undescribed mechanism to sabotage host immune system
6. Baiji Dolphin previously thought extinct spotted in the Yangtze River
7. Oatmeals health claims strongly reaffirmed, science shows
8. Claims of sex-related differences in genetic association studies often not properly validated
9. Mechanism for the in-vivo transport of siRNA
10. AACR hosts Molecular Diagnostics in Cancer Therapeutic Development Meeting
11. CU-Boulder, biotech firm team up on python project in search for human cardiac therapeutics
Post Your Comments:
(Date:10/12/2015)... , Oct. 9, 2015 Research and Markets ... Research, Inc,s new report "Biometrics: Market Shares, Strategies, ... --> --> The ... the Biometrics market segment. Research represents a selection from ... and cogent market materials, with selections made by the ...
(Date:10/12/2015)... -- Research and Markets ( ) has ... Market by Component (Hardware & Software), Product (Scanner ... (Travel & Immigration, Military & Defense, & Others) ... report to their offering. ... worth 3627.90 Million USD by 2020 ...
(Date:10/8/2015)... Oktober 2015 Die Track Group, ... des Bereiches Tracking, hat heute bekannt gegeben, ... Virginias (Department of Corrections – DOC) unterzeichnet ... alle Strafen geliefert werden, die der Behörde ... für den Amerikanischen Kontinent der Track Group. ...
Breaking Biology News(10 mins):
(Date:10/13/2015)... , Oct. 13 2015 Research and ... "US & Europe Markets for Bone Morphogenetic Protein ... to their offering. --> ... that induce the formation of bone after a fracture. ... embryonic development in the formation of the skeleton. There ...
(Date:10/13/2015)... New York , October 13, 2015 ... Growth, Trends, and Forecast 2015 - 2023 " , ... in 2014 and is anticipated to reach US$7.59 bn by 2023, ... 2015 to 2023. --> " Microbiology Culture Market ... - 2023 " , the global microbiology culture ...
(Date:10/13/2015)... ... October 13, 2015 , ... AxioMx Inc. , a ... received a Phase I Small Business Innovative Research (SBIR) grant (1R43GM112204-01A1). This Phase ... Sciences (NIGMS), will fund the development of a technique to rapidly convert single-chain ...
(Date:10/13/2015)... YORK , October 13, 2015 ... acceleration and development company, has entered into a strategic ... York and Paris, France ... --> --> This collaborative arrangement ... highly respected scientific advisory team as well as long ...
Breaking Biology Technology: