Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.

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Articles with the topic: disease-modifying

Important advances in next generation genome editing tools for Huntington's Disease

Important advances in next generation genome editing tools for Huntington's Disease

Mr. Shawn Minnig on October 31, 2016

Recent days have seen a slew of news emerging regarding the use of something called genome editing as a potential therapy for genetic diseases like Huntington's Disease. These approaches, which include exotic sounding tools like zinc finger nucleases and CRISPR/Cas9, differ from more traditional ways reducing the impact of the HD mutation on cells. What's new in this exciting area of research?

Liftoff: First humans treated with gene silencing drugs for HD!

Liftoff: First humans treated with gene silencing drugs for HD!

Dr Jeff Carroll on October 22, 2015

Today brings news that the first Huntington's Disease patients have been successfully dosed with gene silencing drugs targeting the HD gene. These brave volunteers are the first HD patients to ever be treated with drugs designed to attack HD at its root cause, a treatment approach with huge potential. What about this news has us so excited?

Measuring harmful huntingtin protein in the brain’s bath water

Measuring harmful huntingtin protein in the brain’s bath water

Dr Michael Orth on May 18, 2015

Exciting technologies such as gene silencing are being developed for the treatment of Huntington’s disease. Aside from waiting for disease progression to take place, how will we know whether they are working? This has been a major hurdle for HD researchers, but we now have a super-sensitive method to measure the build-up of harmful huntingtin protein in the nervous systems of HD patients.

Could HD be caused by amino acid deficiency?

Could HD be caused by amino acid deficiency?

Dr Jeff Carroll on April 21, 2014

All the proteins in our body are made of tiny chemical building blocks, called amino acids. The internet was recently buzzing about a newly discovered link between one of these amino acids, cysteine, and Huntington's Disease. Is it true, as some headlines suggested, that "Brain Degeneration In Huntington’s Disease Caused By Amino Acid Deficiency"?

Does high-dose creatine "slow the onset" of Huntington's Disease?

Does high-dose creatine "slow the onset" of Huntington's Disease?

Dr Jeff Carroll on February 11, 2014

The results of a new study called PRECREST, investigating whether the nutritional supplement creatine can slow Huntington's disease progression, have just been published. Uniquely, this studied the effects of high-dose creatine supplementation in people carrying the HD mutation, but without clear disease symptoms.

Prana Biotech publishes Huntington's disease animal model data for PBT2

Prana Biotech publishes Huntington's disease animal model data for PBT2

Dr Jeff Carroll on January 14, 2013

The Huntington Study Group and Prana Biotechnology are currently running a clinical trial, Reach2HD, to determine whether the drug PBT2 is effective in HD patients. Now, they've released the preclinical data behind the trial, showing the drug is effective in two animal models of HD.

ASO gene silencing reaches further, lasts longer

ASO gene silencing reaches further, lasts longer

Dr Nayana Lahiri on June 21, 2012

Drugs called anti-sense oligonucleotides, or ASOs, are one way of silencing the gene that causes Huntington's disease. A new publication in the journal Neuron suggests that ASO gene silencing reaches further in the brain than other methods, lasts longer and is safe.

Double success for huntingtin RNAi gene silencing

Double success for huntingtin RNAi gene silencing

Dr Ed Wild on January 24, 2012

Most HD researchers are pretty excited by the idea of 'silencing' the Huntington's disease gene, to reduce production of the harmful huntingtin protein. Two challenges - safety and delivery - are now closer to being solved thanks to collaborative work by academic and industry researchers.

New experiments clarify the role of SIRT1 in HD - or do they?

New experiments clarify the role of SIRT1 in HD - or do they?

Dr Jeff Carroll on January 12, 2012

Biologists are very excited about a protein called SIRT1 - activating it seems to extend life. Could activating this remarkable protein help HD? New experiments in mice suggest that SIRT1-activation might be a good target for HD drugs - but other researchers think the opposite.