Add this to the list of possible applications for the seemingly-magical gene editing technology CRISPR: helping people with neurological disorders edit their brains with pinpoint accuracy to alleviate the symptoms of their disorder.
That’s the potential promise of a new study, out today in the journal Nature Biomedical Engineering. In the study, a team of researchers from the University of Texas and the University of California at Berkeley as well as a Berkeley-based company called GenEdit describe a new process for using gold nanoparticles for the delivery of the Cas9 enzyme that does the editing. They say this technique, first described in a 2017 paper dealing with gene editing in muscle cells, could pose fewer problems than the viruses that serve as CRISPR’s conventional delivery method.
“This paper is the first demonstration that you can do non-viral gene editing in the brain and have local effects,” says Niren Murthy, a Berkeley bioengineer who has been working on new CRISPR delivery vehicles for several years. This kind of localized editing could potentially help treat patients with conditions such as Huntington’s Disease, epilepsy, chronic pain, or even addiction.
She says it might be possible, using this technology, to allow autistic people to treat unwanted symptoms such as repetitive behaviors just like they might with medicine—with the difference that CRISPR changes would likely be permanent.
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