Sentences with phrase «huntingtin rna»
Bates» team showed that a protein usually responsible for the editing of RNA message molecules actually sticks to expanded huntingtin RNA, but not to normal huntingtin RNA.
https://en.hdbuzz.net/
Studying mice, Bates» team has found that the splicing step, where the non-coding gibberish is removed from the RNA message, goes wrong if the huntingtin RNA is expanded, as it is in Huntington's disease.
Until now, it was thought that all the huntingtin RNA in the cell was the full - length version.
This interaction wasn't seen with normal - length huntingtin RNA.
Ionis pharmaceuticals are currently nearing the end of an exciting clinical trial using an ASO that targets the huntingtin RNA.
While everyone agrees that the mutant huntingtin protein is the main cause of harm in Huntington's disease, some researchers think that huntingtin RNA could be an additional source of damage.
Not exact matches
Next, Grima looked at cell death in cultured neurons with a healthy or a mutant form of Huntingtin, or with a mutant form of Huntingtin that was treated with small amounts of an experimental drug called KPT - 350, one that prevents a nuclear export protein, Exportin - 1, from shuttling proteins and RNA out of the nucleus.
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?
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.
17:17 - Using RNA sequencing, Brivanlou has identified 4 new RNA message molecules for huntingtin in embryo cells.
During translation, however, the RNA instructions for extra-long huntingtin can also interact with a helper complex (the assistant chef from our analogy).
Ultimately, cells use this RNA message as instructions for constructing a protein — the huntingtin protein.
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.
It may be that this inappropriate sticking interferes with proper splicing, resulting in the faulty short RNA copy of huntingtin.
In the case of an expanded huntingtin gene, the RNA copy of the recipe is also expanded.
One possible implication of this work is for so - called «gene - silencing» therapies for Huntington's disease, which aim to reduce production of the huntingtin protein, by sticking to its RNA message molecules and telling cells to get rid of them.