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