Sentences with phrase «of the huntingtin gene»

In Huntington's disease, mice carrying the pathologic genetic variant of the huntingtin gene are being used to understand how this genetic lesion causes degeneration of striatal neurons and to develop novel treatments for the illness.

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

Genes that lead to the toxic effects of the huntingtin gene may be silenced by these microRNAs, in particular the miR - 10b - 5p microRNA.

Huntington's disease is caused by the abnormal repetition of a specific DNA sequence at the tail end of the huntingtin gene.

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

Both teams designed zinc finger molecules that would stick to the «CAG tract» of the huntingtin gene, and tell cells not to read the gene.

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?

When a person has too many CAGs in a row near the start of the huntingtin gene, a harmful «mutant» form of the protein is produced based on the instructions in the gene.

Correlation of CAG repeat length between the maternal and paternal allele of the Huntingtin gene: evidence for assortative mating.

It's the extra-long copy of the huntingtin gene that makes neurons sick, because it causes them to produce an extra-long, harmful version of the huntingtin protein.

Drug - makers can build designer molecules with a sequence that will stick to the messenger molecule of the huntingtin gene, but not to other messengers.

One possible explanation is that the mice in the Dragatsis project were unusual to begin with, in that they had only one copy of the huntingtin gene, rather than the usual two.

Everyone has two copies of the huntingtin gene but Huntington's disease is caused by a copy that's extra-long.

Huntington's disease is caused by an unwanted expansion of the huntingtin gene.

Huntington's Disease (HD) is an autosomal dominant inherited disease characterized by an abnormal and unstable expansion in the number of CAG trinucleotide repeats in exon 1 of the huntingtin gene (Group HsDCR, 1993).

Not exact matches

Researchers led by Xiao - Jiang Li, MD, PhD and Shihua Li, MD, at Emory University School of Medicine, used genetically engineered mice in which the huntingtin gene can be deleted, triggered only when the mice are given the drug tamoxifen.

In a nonhuman primate model geneticist Anthony Chan DVM, PhD, and his colleagues at Yerkes developed, rhesus macaques carry a gene encoding a fragment of mutant human huntingtin.

It is caused by a single gene abnormality which leads to the production of a mutant form of a protein called huntingtin (mHtt).

HD is caused by a mutation in the human HTT gene that results in an abnormal expansion and misfolding of the corresponding huntingtin protein.

Not long after the HD gene was isolated, studies led by MacDonald, also a co-author of the current investigation, found that a variation in the number of CAG trinucleotide repeats within the HD gene, which codes for a protein called huntingtin, is the primary determinant of the age at which HD symptoms appear, with a greater number of CAG repeats associated with an earlier symptom onset.

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 zinc finger protein sticks to the DNA of the mutant Huntingtin gene and turns off the gene's expression.

The mutant Huntingtin gene is thought to cause toxic levels of protein to aggregate in the brain.

To further explore nuclear transport's role in Huntington's disease, Grima took lab - grown mouse neurons and used chemical switches to a) turn on both an additional healthy copy of the RanGAP1 gene and a mutant version of Huntingtin; b) just turn on the mutant Huntingtin; or c) just turn on a healthy version of Huntingtin.

These three monkeys carried a gene encoding a fragment of mutant human huntingtin.

Mutations of the huntingtin protein (HTT) gene underlie both adult - onset and juvenile forms of Huntington's disease (HD).

an organism that has had one of its genes altered, for example by adding a long CAG repeat into the huntingtin gene.

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.

R6 / 2 mice have been genetically altered with an abnormal gene that makes them produce a harmful fragment of the mutant huntingtin protein

The disease is linked to a mutation in the Huntingtin gene, which causes a protein of the same name to fold up incorrectly like misshapen origami.

Researchers recently inactivated the huntingtin gene in healthy adult mice of different ages.

Activity of the mutant huntingtin gene was reduced by around 50 % in the brain near the injection site.

More than 36 CAG repeats in the huntingtin gene will always lead to HD symptoms, if a person lives long enough, and longer CAG repeats tend to produce an earlier age of onset.

The expanded CAG sequence in the HD gene results in a huntingtin protein with too many glutamines at the start of it.

Mutations on a single gene, the huntingtin gene, are the cause of Huntington's disease.

The genetic basis of Huntington's disease (HD) is the presence of expanded CAG repeats in the huntingtin gene.

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.

Huntington's disease (HD) is a progressive neurodegenerative disorder caused by a CAG: CTG repeat expansion in exon 1 of the gene that encodes the polyglutamine - containing protein Huntingtin.

With these details of translation in mind, a team of Huntington's disease researchers, led by Susann Schweiger of The Max Planck Institute for Molecular Genetics in Berlin, decided to study how huntingtin proteins are made from genes of different lengths.

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've known for twenty years now that the cause of Huntington's disease is a mutation in the huntingtin gene.

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.

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.

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.

The edits made to the Huntingtin gene during the first four weeks will remain forever, but the eventual deactivation of the Cas nuclease reduces the chances of harmful effects later on.

In HD, a repeated sequence of letters in this gene leads to an extra-long form of huntingtin protein that can wreak havoc in brain cells over long periods of time.

That means it's a single strand of chemically modified DNA, designed to stick to the message molecule from the huntingtin gene.

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.