Sentences with phrase «gene silencing drugs»

In contrast, other gene silencing drugs with names like RNA interference, siRNA or shRNA, need to be injected directly into the substance of the brain and don't spread very far without help.

So far, we've seen gene silencing drugs tested in several different animal models of Huntington's disease, successfully delaying symptom onset or even reversing symptoms.

Since RNA - based gene silencing drugs have been successful so far, why bother with the greater challenge of targeting the DNA of the huntingtin gene itself, especially if it means dealing with virus particles and big, fragile drugs made of protein?

Today brings news that the first Huntington's Disease patients have been successfully dosed with gene silencing drugs targeting the HD gene.

Anti-sense oligonucleotides or ASOs are one type of gene silencing drug, made from a DNA - like chemical.

Now, a major deal between Isis Pharmaceuticals and drug giant Roche promises to support the development of one kind of gene silencing drug, called ASOs, taking them through to clinical trials in patients as quickly and efficiently as possible.

Not exact matches

BOSTON, April 20 (Reuters)- Alnylam Pharmaceuticals Inc said on Friday that Dicerna Pharmaceuticals Inc will pay it $ 25 million in cash and stock to resolve a lawsuit claiming it stole trade secrets about gene - silencing technology used to develop drug treatments.

Today, there are a fair number of gene - silencing drugs in late - stage development and dozens of companies working in the space — and it feels like we could be close to a tipping point.

A version of this article appears in print on, on Page C3 of the New York edition with the headline: Merck Buys Maker of Gene - Silencing Drugs.

So the work goes into the basic science to understand how genes may make you vulnerable, and how drugs may influence what genes are expressed and what genes are silenced.

The researchers treated 63 cancer cell lines (26 breast, 14 colorectal and 23 ovarian) with low - dose 5 - azacitidine (AZA), an FDA - approved drug for myelodysplastic syndrome, that reverses epigenetic changes by stripping off the methyl group that silences the gene.

Being able to detect and measure the amount of mutant huntingtin present in the nervous system will be a valuable way of seeing whether the gene - silencing drug is hitting its target and has the intended effect, lowering the amount of disease causing mHTT protein.

2015 will see the start of the first human clinical trial of a gene silencing or huntingtin - lowering drug, which specifically aims to reduce production of mutant huntingtin in the brains of HD patients.

His current specific interest is the elucidation of novel mechanisms of drug resistance through the discovery / identification of novel gene function (s) and phenotypes using RNAi - mediated gene silencing or cDNA overexpression.

That could be very helpful right now as we are testing a new generation of so - called «gene silencing» drugs that we hope will put the brakes on the condition.

It is, in a sense, the opposite of gene silencing, but it's another proof point that validates the general concept of creating drugs that mute or amplify defective genes.

Less than a decade after a powerful gene - silencing method — RNA interference, or just RNAi — was discovered, the field's pioneers have not only won the 2006 Nobel Prize in Physiology or Medicine but have also helped launch an entirely new class of drugs into human clinical trials.

And many companies began to crumble: While gene silencing was simple in worms, mammals proved to be far more complex — and safe drug delivery emerged as a major problem.

18 Drugs Aim to Silence Bad Genes RNA interference helped launch an entirely new class of drugs into triaDrugs Aim to Silence Bad Genes RNA interference helped launch an entirely new class of drugs into triadrugs into trials...

By synthesizing a nanoparticle that releases its siRNA cargo only after it enters targeted cells, Dr. Tariq M. Rana and colleagues showed in mice that they could deliver drugs that silenced the genes they wanted.

The researchers at Bonn University and their colleagues from the Federal Institute of Drugs and Medical Devices (BfArM) and from the University of Regensburg worked with so - called transgenic and knockout mice in whom the gene for micro-RNA 155 was either increased or silenced.

Researchers have collected information on which genes are activated or silenced in certain diseases and by certain drugs for many years.

Drugs capable of activating silenced genes improve survival and growth outcomes in a mouse model of Prader - Willi syndrome (PWS), a rare and incurable childhood disease, according to a study funded by the National Institutes of Health (NIH).

Toni Bedalov investigates drug candidates for anticancer therapy in breast - cancer malignancies, illuminates the molecular process of gene silencing

ALS can be caused by a mutation in a protein called SOD1, and the antisense drug effectively silences the gene that codes for this mutant protein — found in the cells of patients with inherited forms of ALS.

Features a clinical trials roundup, an exclusive interview with Prof Sarah Tabrizi about the first trial of a huntingtin lowering «gene silencing» drug, and a surprise for EHDN president Prof Bernhard Landwehrmeyer.

It would be a form of «huntingtin lowering» or «gene silencing», but one that doesn't reply on DNA - like or RNA - like drugs, which are difficult to deliver to the brain.

«We have found that the same genes responsible for tamoxifen resistance in our animals are also turned off in human breast cancer cells that do not respond to the drug,» she says «Because these genes were epigenetically silenced — meaning they were not irreversibly altered, just switched off — it was possible to turn them back on.

Many people, including HDBuzz, have referred to these as «gene silencing» drugs.

Although treating atherosclerosis with the drug decitabine is not a viable option clinically, Jo's team was able to find several genes that are silenced by disturbed blood flow and that need DNA methylation to stay shut off.

In this episode, Dr. Carlos Faraco speaks with Dr. Don Cleveland of the University of California San Diego regarding his work on drug - based gene silencing therapies.

Cleveland and the members of his lab use these therapies, also known as designer DNA drugs, to silence genes involved in the development of various neurodegenerative diseases such as Alzheimer's, Huntington's and Lou Gehrig's disease.

A new gene - silencing drug gives hope of a future where keeping LDL cholesterol levels low is as simple as a small injection from your GP once or twice a year (Credit: < a href ="http://depositphotos.com/43422131/stock-photo-cholesterol-in-a-vein.html" rel="nofollow"> beawolf / Depositphotos )

Silver Nanoscale Antisense Drug Delivery System for Photoactivated Gene Silencing, ACS Nano (2013).

Researchers will have to bear in mind that some of the detrimental huntingtin protein may come from a shorter RNA message, which may be missed by some gene - silencing drugs.

That's a surprisingly difficult thing to do, because the place where the mutant gene is different isn't necessarily the best place for a silencing drug to stick.

He also runs clinical trials of new treatments in HD including «gene silencing» drugs.

Mipomersen (Kynamro) reduces low - density lipoprotein (LDL) cholesterol by silencing a gene that produces apolipoprotein B - 100, a building block of LDL; the drug can treat familial hypercholesterolemia, an inherited disease that may cause heart attacks at an early age.

Isis Pharmaceuticals and Roche have announced a multi-million dollar deal to support the development of «gene silencing» drugs to human trials.

The drug — which also involves researchers from Isis Pharmaceuticals in Carlsbad — basically silences the ill effects of the bad gene.