Navigation Links
New therapeutic strategy could target toxic protein in most patients with Huntington's disease
Date:4/9/2009

. We don't know why," Zamore said. That meant a single siRNA could shut off expression in the mutant Huntingtin gene while leaving cells' healthy Huntingtin genes intact -- in almost half of all U.S. and European Huntington's patients.

"The most exciting part of the study was finding one siRNA that clearly is the top candidate for a clinical trial, where the patient population is predicted to be sufficiently large that it merits the development of a drug you could take into trial," Zamore said.

"That takes away the biggest worry we had, which was that the number of siRNAs we would have to test in order to have impact on the disease would be too large, and as a consequence the FDA would never approve any trial," he continued.

By adding an siRNA against one of two other common SNPs, Zamore says gene silencing could be effective in 75 percent of patients with Huntington's disease in the U.S. and Europe. Although the group found other SNPs, targeting more of them failed to increase the number of patients who could be helped, he said.

The next problem became developing siRNAs that could discriminate between target mRNAs and non-target. "That turned out to be frustratingly difficult," Zamore said. In tests of human cells, the siRNA sometimes sliced up the disease RNA, as it should. But sometimes it destroyed the normal Huntingtin RNA as well. To prevent this error, Zamore and his colleagues changed one more nucleotide base on the siRNA. Now, the silencing RNA was different from the healthy mRNA by two nucleotides, making it less likely to grab the good RNA.

Further research in mice will examine the efficacy of the siRNA tool. "The siRNA has to be sufficiently stable, and has to get into the right cells, and has to discriminate between the two (genes). It's incredibly expensive work," he said.

Zamore acknowledges that even with this progress, they're a long way from a treatment for Huntington's. "T
'/>"/>

Contact: Jennifer Michalowski
michalow@hhmi.org
301-215-8576
Howard Hughes Medical Institute
Source:Eurekalert

Page: 1 2 3 4

Related biology news :

1. Using evolution, UW team creates a template for many new therapeutic agents
2. The endocannabinoids: Functional roles and therapeutic opportunities
3. Potential new therapeutic molecular target to fight cancer
4. Galapagos and Cystic Fibrosis Foundation Therapeutics announce drug discovery collaboration
5. Methadone even at therapeutic levels can kill
6. Nucleonics initiates hepatitis B clinical trial with expressed interfering RNA therapeutic
7. Synthetic molecules may be less expensive alternative to therapeutic antibodies, researchers find
8. New technology enhances therapeutic potential of cord blood stem cells
9. Novel therapeutic strategy for colon cancer treatment
10. TG2 identified as potential therapeutic target in chemo-resistant ovarian cancer
11. Michael J. Fox Foundation awards $2.4M for validation of therapeutic targets for Parkinsons
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/27/2014)... that nicotine and cotinine, a metabolite of nicotine, can ... in smoke. , The carcinogen 4-(methylnitrosamino)-1-(3-pyridyl)-1-butanone or NNK is ... ends up in the tobacco smoke. Once inhaled, it ... is activated by a variety of enzymes called Cytochrome ... nicotine can partially interfere with the activation of NNK, ...
(Date:7/27/2014)... might have survived the asteroid strike that wiped them ... later in history, scientists say. , A fresh study ... helped palaeontologists to build a new narrative of the ... They found that in the few million years before ... was experiencing environmental upheaval. This included extensive volcanic activity, ...
(Date:7/27/2014)... to date, researchers from the U.K. provide national, regional, ... virus (HCV). Findings published in Hepatology, a journal of ... indicate that genotype 1 is the most prevalent worldwide, ... reside in East Asia. Genotype 3, at just over ... by genotypes 2, 4, 6, and 5. , ...
Breaking Biology News(10 mins):Nicotine found to inhibit DNA-strand break caused by a certain carcinogen in smoke 2Dinosaurs fell victim to perfect storm of events, study shows 2Hepatitis C virus genotype 1 is most prevalent worldwide 2
... July 11, 2013 /PRNewswire-iReach/ -- Chris Christie , Governor ... lost a significant amount of weight after undergoing lap band ... in USA Today, Governor Christie was inspired to have the ... clothes no longer fit. (Photo: http://photos.prnewswire.com/prnh/20130711/CG45941 ) ...
... are obese and hope to be a father, here,s ... may inherit your waistline or metabolic disorders. That,s because ... those who did not show any signs of diabetes, ... daughters. Sons don,t entirely dodge this genetic bullet ...
... experimental study in rats has shown that consuming choline, ... eggs and chicken or beef liver, soy and wheat ... study, conducted by scientists at the University of Granada ... York (United Kingdom), has revealed that choline is directly ...
Cached Biology News:Chris Christie, Governor of New Jersey, Slims Down after Having Lap Band Surgery 2Obese dads pass on predisposition to obesity and metabolic disorders to their kids 2Choline intake improves memory and attention-holding capacity 2
(Date:7/26/2014)... July 26, 2014 Canada Endoscopy ... market data on the Canada Endoscopy Visualization Systems ... US dollars, volume (in units) and average prices ... Endoscopy Visualization Systems, Mid-Range Endoscopy Visualization Systems and ... at http://www.marketresearchreports.biz/analysis/213254 . , The report also ...
(Date:7/25/2014)... July 26, 2014 "Ischemic Stroke ... 2014" provides data on the Ischemic Stroke and ... elemental information and data relating to the clinical ... View full global Ischemic Stroke Research Report 2014 ... overview of the trial numbers and their recruitment ...
(Date:7/25/2014)... 25, 2014 Amgen (NASDAQ: AMGN ... a $0.61 per share dividend for the third quarter ... 5, 2014, to all stockholders of record as of ... About Amgen Amgen is committed to unlocking ... illnesses by discovering, developing, manufacturing and delivering innovative human ...
(Date:7/25/2014)... MA -- Trillions of bacteria live in each person,s ... help digest food and stave off harmful infections, but ... , To help shed light on the role of ... associate professor Eric Alm recently tracked fluctuations in the ... year. The findings, described in the July 25 issue ...
Breaking Biology Technology:Canada Endoscopy Visualization Systems Market Outlook to 2020: MarketResearchReports.Biz 2Canada Endoscopy Visualization Systems Market Outlook to 2020: MarketResearchReports.Biz 3Canada Endoscopy Visualization Systems Market Outlook to 2020: MarketResearchReports.Biz 4Canada Endoscopy Visualization Systems Market Outlook to 2020: MarketResearchReports.Biz 5Ischemic Stroke and Cerebral Stroke Global Clinical Trials Review H2, 2014: MarketResearchReports.Biz 2Ischemic Stroke and Cerebral Stroke Global Clinical Trials Review H2, 2014: MarketResearchReports.Biz 3Amgen Announces 2014 Third Quarter Dividend 2Amgen Announces 2014 Third Quarter Dividend 3Monitoring the rise and fall of the microbiome 2Monitoring the rise and fall of the microbiome 3
... nanomaterial developed at Rensselaer Polytechnic Institute could enable the ... electric automobiles, as well as batteries for laptop computers, ... material, dubbed a "nanoscoop" because its shape resembles a ... can withstand extremely high rates of charge and discharge ...
... Corporation (Nasdaq: SIAL ) will be presenting at ... 10th at 10:00 AM PT in San Francisco, CA. Interested ... available at http://investor.sigmaaldrich.com/ .  Users can click the ... About Sigma-Aldrich: Sigma-Aldrich is a leading Life ...
... Rx Communications Group, LLC will host a panel discussion ... with financial as well as trade journalists. The event ... in San Francisco on Tuesday, January 11, 2011 from ... focus on media interview preparation and understanding the differing ...
Cached Biology Technology:'Nanoscoops' could spark new generation of electric automobile batteries 2'Nanoscoops' could spark new generation of electric automobile batteries 3Rx Communications Group to Host Panel Discussion for Biotechnology Executives on Communicating with the Media 2Rx Communications Group to Host Panel Discussion for Biotechnology Executives on Communicating with the Media 3
Mouse monoclonal antibody raised against a partial recombinant AAAS. NCBI Entrez Gene ID = AAAS...
... Cosmid Cloning Kit enables you to prepare, ... unbiased primary cosmid library of genomic DNA ... sublibraries. High efficiency cosmid library construction is ... protocols. First, genomic DNA is randomly sheared ...
Mouse monoclonal antibody raised against a partial recombinant EBI3. NCBI Entrez Gene ID = EBI3...
... VersaDoc MP 4000 System is a ... system capable of supporting a wide ... chemiluminescence, densitometry, and chemiflourescence. Red, ... imaging of multiplexed fluorescent samples, reducing ...
Biology Products: