Sentences with phrase «targeted huntingtin»

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.

The Swiss team's cool idea was to make a CRISPR / Cas machine that targets the huntingtin gene — but with an extra CRISPR sequence that also makes the Cas nuclease target its own DNA.

Ionis pharmaceuticals are currently nearing the end of an exciting clinical trial using an ASO that targets the huntingtin RNA.

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.

The zinc finger protein works by targeting the mutant copies of the Huntingtin gene, repressing its ability to express and create harmful proteins.

«We don't know exactly how the mutant Huntingtin gene causes the disease, so the idea is that targeting the gene expression cuts off the problem at its source — preventing it from ever having the potential to act,» said Dr Isalan.

Zeitlin says the study may also help researchers find new targets for drugs against Huntington's — such as compounds that block the clumping of abnormal huntingtin.

An approach to treating HD that uses targeted molecules to tell cells not to produce the harmful huntingtin protein

A quirk of the huntingtin gene might be helpful when it comes to avoiding these «off - target effects».

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?

Another option may be the pursuit of «allele - specific» therapies, those that specifically target mutant huntingtin while leaving the normal form intact.

Huntingtin, the protein that causes Huntington's disease, has been an elusive target.

Rigorous experiments in animals have given way to clinical trials of drugs that target the HD gene in people, and there are more techniques for decreasing or eliminating huntingtin on the horizon.

It's the world's first trial in patients to slow, stop, prevent or reverse HD, using a method that targets the root cause of the disease, a gene producing a protein called huntingtin.