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Important advances in next generation genome editing tools for Huntington's Disease
Work with genome editing techniques (zinc fingers and CRISPR) brings these tools closer to use in HD clinical trials
October 31, 2016
A step forward for gene editing: CRISPR-Cas9 and HD
Evolving CRISPR-Cas9 techniques can now be used to edit the HD gene in a living mouse brain.
July 26, 2017
Unpacking recent gene therapy press
Voyager Therapeutics is shifting towards a new technology to deliver gene therapy and away from a planned HD clinical trial. But this could lead to less invasive drugs in the long run, and many other companies are working on HD gene therapies.
August 16, 2021
Gene silencing for HD: the story so far
Is gene silencing as exciting as it sounds for HD, and what might the future hold?
March 22, 2011
'Floating' gene-silencing drugs into the brain with exosomes
Exosomes – tiny bubbles made by cells – could be used to get gene silencing drugs from the bloodstream into the brain
May 19, 2011
Gene silencing takes a targeted step forward
Targeting the mutant Huntington's disease gene for silencing, while leaving the healthy gene untouched
October 07, 2011
Safety trials add crucial piece to gene silencing jigsaw
Three trials of drugs to 'silence' the HD gene in monkeys show they hit the target safely. Human trials coming soon.
November 14, 2011
Double success for huntingtin RNAi gene silencing
2 bits of good news for RNAi gene silencing in HD: it's safe over six months, and a way to treat bigger brain areas
January 24, 2012
Could mesenchymal stem cells deliver gene silencing drugs?
Stem cells can deliver HD gene silencing drugs into cells in a dish. Do we need another delivery technology?
January 31, 2012
ASO gene silencing reaches further, lasts longer
Silencing the Huntingtin gene using ASO drugs reaches further, lasts longer and is safe. Human trial soon?
June 21, 2012
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