Navigation Links
Previous claims of siRNA therapeutic effects called into question by report in human gene therapy
Date:9/2/2008

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 www.liebertpub.com/hum

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
vcohn@liebertpub.com
914-740-2156
Mary Ann Liebert, Inc./Genetic Engineering News
Source:Eurekalert

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:
*Name:
*Comment:
*Email:
(Date:8/23/2017)... -- The general public,s help is being enlisted in what,s thought to be ... on the human body –and are believed to affect health.  ... The Microbiome Immunity Project is the largest study ... gut. The project's goal is to help advance scientific knowledge of the ... ...
(Date:6/30/2017)... Today, American Trucking Associations announced Seeing ... and eye tracking software, became the newest member ... "Artificial intelligence and advanced sensing algorithms ... driver,s attentiveness levels while on the road.  Drivers ... fatigue and prevent potential accidents, which could lead ...
(Date:5/23/2017)...  Hunova, the first robotic gym for the rehabilitation and functional motor ... Genoa, Italy . The first 30 robots will be ... USA . The technology was developed and patented at the ... spin-off Movendo Technology thanks to a 10 million euro investment from entrepreneur ... ...
Breaking Biology News(10 mins):
(Date:10/12/2017)... ... October 12, 2017 , ... AMRI, a global contract research, ... improve patient outcomes and quality of life, will now be offering its impurity ... to new regulatory requirements for all new drug products, including the finalization of ...
(Date:10/11/2017)... LINDA, CA (PRWEB) , ... October 11, 2017 ... ... to upregulate any gene in its endogenous context, enabling overexpression experiments and avoiding ... (CRISPRa) system with small RNA guides is transformative for performing systematic gain-of-function studies. ...
(Date:10/11/2017)... ... October 11, 2017 , ... Proscia Inc ... hosting a Webinar titled, “Pathology is going digital. Is your lab ready?” with ... adoption best practices and how Proscia improves lab economics and realizes an increase ...
(Date:10/11/2017)... ... October 11, 2017 , ... Disappearing forests and increased emissions are the ... million people each year. Especially those living in larger cities are affected by air ... one of the most pollution-affected countries globally - decided to take action. , “I ...
Breaking Biology Technology: