Ionis has built a sort of model that enables them to make predictions about the relationship
between huntingtin lowering in the spinal fluid and in brain tissues.
Scientists have long assumed that the disorder arose from the interaction
between huntingtin and so - called caspases, enzymes involved in the routine breakdown of proteins.
Not exact matches
Yuan's team exposed human brain cells to Congo red
between 6 and 48 hours after they began producing
huntingtin.
Huntington's patients have a mutant form of a protein called
huntingtin that contains
between 36 and 120 repeats of the amino acid glutamine, where unaffected individuals have just a couple dozen repeats.
Here's what we know so far about the trial, the company, the drug and the friendship
between copper and the mutant
huntingtin protein.
Correlation of CAG repeat length
between the maternal and paternal allele of the
Huntingtin gene: evidence for assortative mating.
Edited to clarify the difference
between complete reduction of
huntingtin in the mouse experiments, as opposed to partial reduction by
huntingtin - lowering drugs
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.
Wild - type
huntingtin participates in protein trafficking
between the Golgi and the extracellular space.
This article was updated on 26th September 2017, in response to reader feedback, to clarify the important differences
between total
huntingtin removal in mice and partial reduction by
huntingtin lowering drugs.