Sentences with phrase «huntingtin gene»

In the United States approximately 10 per 100,000 have the faulty huntingtin gene and live either waiting for symptoms to begin or live / struggle with the disease.

The CAG 140 KI mouse model carries a chimeric mouse / human exon 1 containing around 125 CAG repeats and the human polyproline region inserted in the murine huntingtin gene.

Spontaneous expansion of the CAG repeat stretch in the CAG 140 KI mouse model, which carries a chimeric mouse / human exon 1 containing around 125 CAG repeats and the human polyproline region inserted in the murine huntingtin gene (Menalled et al., 2003, Hickey et al., 2008), has recently led to a new KI line that carries around 190 CAG repeats (CAG 190 KI) in a congenic C57Bl / 6J background.

In the case of an expanded huntingtin gene, the RNA copy of the recipe is also expanded.

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).

The 120 repeat R6 / 2 mouse model of HD expresses a human transgene containing exon 1 of the mutant huntingtin gene and faithfully replicates many of the symptoms of the disease, including progressive loss of body weight, marked impairments in cognition, and severe motor deficits.

The instruction set for making the huntingtin protein - the huntingtin gene - is stored in every cell and is made of DNA.

But in mice engineered to carry an expanded huntingtin gene, the first non-coding region was not removed properly.

The huntingtin gene is very long - one of the longest genes we have - and stores the recipe for a very big protein.

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

It was initially called ISIS - 443139, but lately it's been going by IONIS - HTTRx or just HTTRx — a combination of HTT, the abbreviation scientists use for the huntingtin gene, and Rx, a symbol used in the pharmaceutical industry meaning a treatment or prescription.

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

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.

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.

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

That means it first deactivates the huntingtin gene, and then about four weeks later the genome editing system shuts down.

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.

The KamiCas9 genome editing system first deactivates the huntingtin gene, and then about four weeks later it shuts itself down

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.

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

Two CMBN groups have identified a DNA repair gene that modify somatic and germline CAG trinucleotide repeat instability in the Huntingtin gene.

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.

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

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

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

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.

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?

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.

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

Any effects were small compared with the desirable action on the mutant huntingtin gene.

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.

We know for sure that the mutation in the huntingtin gene is the ultimate reason why people get HD.

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

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.

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

Adult mice don't need the gene that, when mutated in humans, causes the inherited neurodegenerative disorder Huntington's disease. The finding suggests that treatment strategies for Huntington's that aim to shut off the huntingtin gene in adults — now in early clinical stages — could be safe.

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

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

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

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

That means a healthy parent whose huntingtin gene encodes proteins with 35 repeats may produce a child with 36 repeats.

«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.

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

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

Anyone with more than 36 repeats in the huntingtin gene will develop Huntington's disease.

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.

When the huntingtin gene is deleted at an age older than four months, these mice appeared to stay healthy, despite having lost their huntingtin genes in cells all over their bodies.

The finding suggests that treatment strategies for Huntington's that aim to shut off the huntingtin gene in adults — now in early clinical stages — could be safe.

The huntingtin gene encodes a large scaffold protein, with many interaction partners, which is thought to be involved in intracellular trafficking.

The huntingtin gene is essential for embryonic development, and scientists have already shown that if mouse embryos don't have it at conception, they die in utero.