Sentences with phrase «target mutant huntingtin»
Another option may be the pursuit of «allele - specific» therapies, those that specifically target mutant huntingtin while leaving the normal form intact.
https://en.hdbuzz.net/ Not exact matches
Being able to detect and measure the amount of mutant huntingtin present in the nervous system will be a valuable way of seeing whether the gene - silencing drug is hitting its target and has the intended effect, lowering the amount of disease causing mHTT protein.
The zinc finger protein works by targeting the mutant copies of the Huntingtin gene, repressing its ability to express and create harmful proteins.
«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.