How
HD gene silencing is supposed to work.
Not exact matches
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.
Today brings news that the first Huntington's Disease patients have been successfully dosed with
gene silencing drugs targeting the
HD gene.
One of the most exciting avenues of
HD research is huntingtin lowering (also known as
gene silencing), which aims to reduce levels of the huntingtin protein in cells.
The current ASO trial in humans is a huntingtin lowering, or
gene silencing therapy, which works to disable both copies of the
HD gene in short bursts.
She leads a team at the UCL Huntington's Disease Centre that is currently conducting a trial of a novel «
gene silencing» treatment for
HD, sponsored by Ionis Pharmaceuticals.
Like many scientists, we think that
gene silencing is the most promising approach to developing meaningful therapies for
HD families, and it's gratifying that large companies are willing to make a large financial investment in the therapy.
12:10 - David Corey is working on new ways to «
silence» the mutant
HD gene.
He also runs clinical trials of new treatments in
HD including «
gene silencing» drugs.
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?