It's the kind of stuff that seems to come from science fiction movies; a technology that allows us to edit the human genome and target diseases that previously we would have never dreamed it could be possible. It has only been five years that CRISPR technology has been in existence and yet, according to the New Scientist, there are approximately twenty human trials that have either begun or are about to begin using the technology, most of which are in China. While the technology is basically touted as being able to treat anything and everything from cancer to muscular dystrophy, what hope does it offer for Huntington's Disease and Juvenile Huntington's Disease?
First, we have to address a problem with the viral delivery system associated with CRISPR. It can take a few weeks for the process to shut down in your body which could mean that the longer it keeps doing what it does, the more chances it has of doing something it was not meant to do, which would mean adverse effects to the patient. However, Nicole Deglon of Lausanne University Hospital in Switzerland, along with her colleagues have developed a "kamikaze" CRISPR that not only prevents the protein from lingering too long but also disables the target gene. Last month (September, 2017), her team conducted a mouse study that targeted the gene that causes Huntington's Disease and they found they were able to disable the gene in 65 percent of the cells in key areas of the animals brains.
According to the study published on Cell.com, the Swiss team is using the technology for use in treating neurodegenerative diseases and sees Huntington's Disease as a major public health issue especially now with an aging population. The study mentions RNA silencing tools that show we can decrease the expression level of the mutant HTT and that last for the long term. However, RNA silencing only provides a partial suppression. Complete and permanent HTT disruption would be preferable and actually can be achieved with genome-editing technologies.
These reports give us reason to hope for not only a treatment, which most of us would gladly welcome, but also a permanent fix. For any technology to go from inception to twenty human trials in five years is astounding and offers real hope to millions of people who suffer from neurodegenerative diseases.