Sentences with phrase «not mutant huntingtin»

Remember we're talking about normal, not mutant huntingtin here.

Not exact matches

The VCP inside mitochondria only interacted with mutant, but not healthy huntingtin protein.

The international team of scientists from University College London, IRBM Promidis, University of British Columbia, and CHDI Foundation developed a new ultra-sensitive test using the Singulex SMC Technology Erenna Immunoassay system that is able to detect mutant huntingtin in the cerebrospinal fluid (CSF) of HD patients, including some who carry the HD mutation but have not yet developed symptoms.

«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.

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.

Note: these mice don't produce toxic mutant huntingtin protein.

First things first - the news that IONIS - HTTRx lowers the mutant huntingtin protein is great - but it is not a cure.

That's not what we expect when patients are given huntingtin - lowering drugs, which might produce around 50 - 75 % reductions in the mutant and healthy 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.