Sentences with phrase «if huntingtin»
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.
https://en.hdbuzz.net/ Not exact matches
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.
«The challenge that remains is if there are many proteins interacting with the huntingtin protein, we can not easily determine which are relevant for disease and which are not,» said Erich Wanker from Max Delbrück Center for Molecular Medicine and corresponding author of the study.
In March Chris Ross and his colleagues reported that cells bearing mutant huntingtin could survive if they produced extra CBP.
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.
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.
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?
Emphasising their desire to move the drug forward, in a separate announcement made directly to the HD community, Ionis said «Upon study completion, the next step for this program will be to conduct a study to investigate if decreasing mutant huntingtin protein with IONIS - HTTRx can slow the progression of this terrible disease.»
Years ago, they realized that HD was a perfect fit for their technology, because we know that if, in animals, we reduce the levels of the huntingtin protein in the brain, we improve their HD - like symptoms.
If we could do it in people, it'd mean that extra-long or «mutant» huntingtin would never even have a chance to make neurons sick.
But understanding how the brain develops and works, and «knowing the enemy» - the mutant huntingtin protein and its damaging effects - are both crucial if we are going to safely and rapidly develop the treatments we're all working towards.
For example, if we block mutant huntingtin production in cells or animals with «gene silencing» techniques, how can we confirm that this treatment actually does what it's supposed to do in the brains of patients with HD?