Sentences with phrase «huntingtin production»
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?
https://en.hdbuzz.net/ Not exact matches
It is caused by a single gene abnormality which leads to the production of a mutant form of a protein called huntingtin (mHtt).
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.
Juliette Gafni, Theodora Papanikolaou, Francesco DeGiacomo, Jennifer Holcomb, Sylvia Chen, Menalled L.; Kudwa A.; Jon Fitzpatrick, Miller S.; Ramboz S.; Pasi Tuunanen, Kimmo Lehtimaki, Xiangdong William Yang, Seung Kwak, Larry Park, David Howland, Hyunsun Park, and Lisa Ellerby Caspase - 6 activity in a BAC HD mouse modulates steady state levels of mutant huntingtin protein but is not necessary for production of a 586 amino acid proteolytic fragment.
This leads to the production of an abnormally long version of the huntingtin protein.
This stopped the production of multiple harmful huntingtin proteins.
Current huntingtin - lowering drugs, called ASOs, are also given as individual doses separated by several weeks, with normal protein production expected to bounce back somewhat in between.
In July 2015, the most exciting drug trial so far in Huntington's disease began - one in which an ASO designed to reduce production of huntingtin protein was actually delivered to people with HD.
It's important to emphasise that this technique completely shuts off production of the huntingtin protein.
Gafni J, Papanikolaou T, DeGiacomo F, Holcomb J, Chen S, Menalled L, Kudwa A, Fitzpatrick J, Miller S, Ramboz S, Tuunanen P, Lehtimaki K, William YX, Kwak S, Park L, Howland D, Park H, Ellerby L.Caspase - 6 activity in a BACHD mouse modulates steady - state levels of mutant huntingtin protein but is not necessary for production of a 586 amino acid proteolytic fragment.
The trial was designed to test the safety of the drug and whether or not the drug could do what it was designed to do - reduce the production of the huntingtin protein.
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.