Sentences with phrase «huntingtin lowering»

This article was updated on 26th September 2017, in response to reader feedback, to clarify the important differences between total huntingtin removal in mice and partial reduction by huntingtin lowering drugs.

The huntingtin lowering ASO trial is still in early safety stages, but the approach has shown promise so far.

Since the goal of huntingtin lowering therapies like IONIS - HTTRx is to reduce the amount of the huntingtin protein in vulnerable brain cells, in theory this gives us a great way to tell whether the drug is doing what it's meant to do.

So, increasingly, you'll hear us call them «huntingtin lowering» drugs.

However, we're excited and optimistic about huntingtin lowering treatments, and WAVE's latest refinement looks like it could be a an exciting advance.

The current ASO trial in humans is a huntingtin lowering, or gene silencing therapy, which works to disable both copies of the HD gene in short bursts.

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.

This approach is being used by WAVE Life Sciences, a company working on developing allele - specific huntingtin lowering drugs for HD.

After huge leaps forward in recent years, we're edging ever closer to human trials of huntingtin lowering or «gene silencing» as a potential treatment for Huntington's Disease.

An exciting «huntingtin lowering» trial is currently underway using a drug called an antisense oligonucleotide (ASO) to reduce the amount of huntingtin protein in brain cells.

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.

We're excited about this novel approach to huntingtin lowering therapies, but these are early days and we've got a long way to go to show they're safe and effective in people.

One of the most exciting avenues of HD research is huntingtin lowering (also known as gene silencing), which aims to reduce levels of the huntingtin protein in cells.

Ionis has built a sort of model that enables them to make predictions about the relationship between huntingtin lowering in the spinal fluid and in brain tissues.

This suggests that huntingtin lowering in brain tissue might be quite high.

Not exact matches

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.

The test, called a «single molecule counting assay», combines fluorescent antibodies with a laser detection chamber to count individual molecules of mutant huntingtin with a very low detection threshold.

In an announcement likely to stand as one of the biggest breakthroughs in Huntington's disease since the discovery of the HD gene in 1993, Ionis and Roche today announced that the first human trial of a huntingtin - lowering drug, IONIS - HTTRx, demonstrates that it reduces mutant huntingtin in the nervous system, and is safe and well - tolerated.

If what you've read here about zinc finger technology reminds you of «gene silencing» or «huntingtin - lowering» methods for treating Huntington's disease, give yourself a gold star.

Most huntingtin - lowering attempts so far have tried to «shoot the messenger» rather than attacking the source of the message - the DNA itself.

First things first - the news that IONIS - HTTRx lowers the mutant huntingtin protein is great - but it is not a cure.

There are many ways of achieving the goal of lowering mutant huntingtin, expected to start new trials soon or already in early trials.

Huntingtin - lowering drugs are made from molecules similar to DNA, the stuff that makes up our genes.

Later, maybe in early 2018, there may be a separate announcement about some of the more experimental results of the safety trial - things like whether HTTRx lowered the huntingtin level in the spinal fluid.

HDBuzz Editor - in - Chief Ed Wild is a consultant to WAVE Life Sciences on their huntingtin - lowering programme, and to Ionis Pharmaceuticals and Roche on theirs.

Some of these approaches to attack mutant huntingtin also reduce levels of the normal protein, including the Ionis huntingtin - lowering drug.

Ionis» trial, led in partnership with Prof Sarah Tabrizi of University College London, was the first time a targeted huntingtin - lowering drug had been tested in humans.

Edited to clarify the difference between complete reduction of huntingtin in the mouse experiments, as opposed to partial reduction by huntingtin - lowering drugs

Importantly, both studies suggest that we need to continue our current cautious approach when lowering normal huntingtin in human studies.

Current huntingtin - lowering drugs, called ASOs, are also given as individual doses separated by several weeks, with normal protein production expected to bounce back somewhat in between.

That's not what we expect when patients are given huntingtin - lowering drugs, which might produce around 50 - 75 % reductions in the mutant and healthy protein.

We will need to continue to use caution when removing or lowering normal huntingtin in human studies

The therapy we're most excited about for Huntington's disease is called huntingtin - lowering.

The safety data may come first, but information about whether treatment with HTTRx lowered the level of huntingtin protein in the spinal fluid — a much - anticipated «biomarker» outcome — may take a little longer to materialise.

That's much more dramatic than what happens in a huntingtin - lowering treatment trial.

Doing that lowered levels of the protein for which the gene is a recipe: mutant huntingtin.

In the huntingtin - lowering trial that is currently underway, the treatment is reversible, and participants are being carefully monitored for safety.

We know that lowering the level of mutant huntingtin protein in HD mouse models significantly improves symptoms reminiscent of HD, providing hope that similar treatments in people may be effective.

As the short - term administration periods for the huntingtin - lowering drug are extended, clinicians will continue to be watchful and to collect data that is essential to determine whether the treatment is safe and effective.