An international group of scientists has completed some work on the HD mouse model and demonstrated it is possible to ‘edit’ the genetic change that causes HD. The technique is called Zinc Finger Protein Transcription Factors (ZFPs) – essentially microscopic molecular machines capable of snipping out pieces of DNA. Our
Research Updates
Huntington’s disease goes viral as UniQure inches ahead in gene therapy race
This information is provided from a recent blog published by HDBuzz. To view more of their articles or subscribe directly to news published by HDBuzz you can find more information on their website: www.en.hdbuzz.net/ By Dr Jeff Carroll January 29, 2019 Edited by Dr Ed Wild Dutch-American company uniQure has received approval from the US drug regulator the FDA, to begin the first ever gene therapy trial in Huntington’s Disease. Their
Is CRISPR the future of HD treatment?
As the current work by WAVE Life Sciences and IONIS/Roche into Huntingtin Lowering Therapy (gene silencing) unfolds and we wait for the outcome of clinical trials, scientists are already working on new technologies that might treat Huntington’s Disease. One of those is called CRISPR; it is a method for cutting
A cautious welcome for the latest results from IONIS Phase One Trial
So, after posting an article this morning about the inevitable lull since IONIS released the results of its phase one trial, they have now issued a press release providing additional results from their analysis of the phase one study. You can see it by following this link http://ir.ionispharma.com/news-releases/news-release-details/new-data-ionis-htt-rx-phase-12-study-demonstrates-correlation Let’s do a
Molecule behind Huntington’s disease found to be cancer-killing “super assassin”
For several years, scientists have noticed an unexplained reduced incidence of cancer in patients suffering from the devastating hereditary neurodegenerative condition Huntington’s disease. Now a team at Northwestern Medicine has uncovered how the disease could be inadvertently killing cancer cells, and how this process could be harnessed for a new
Protein that causes HD mapped!
HDBuzz-256-en Fascinating article on HD Buzz telling the story of how the HD protein – one of the largest in the human body – has finally been mapped. As they say, if you know your enemy, you stand a much better chance of defeating it!
Grab a coffee and set aside twenty minutes!
Watch this fantastic video by Charles Sabine giving an overview of HD research.
A wave of new ideas?
There has been a great deal of focus over the last few months on the work of IONIS pharmaceuticals who are developing a gene silencing drug (currently known as IONIS HTTrx); this is largely because they were the first to complete a successful phase one clinical trial and announce very
IONIS release data from phase one gene silencing trial
In case you missed the webcast from IONIS, here are the main points: 49% mean reduction in the faulty protein (40%-60%), but levels still declining at six months after start of treatment. IONIS believe, based on the work they did prior to this trial in animal models that this will