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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. And the new paper
offers an almost-unheard of application for CRISPR: editing inside the adult brain.
“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.
In this study, the researchers used CRISPR-Gold to edit the brains of mice with fragile X syndrome,
a genetic condition that is correlated with autism spectrum disorder (ASD). While fragile X syndrome
is a genetic disorder that can be diagnosed in genetic testing, clinicians diagnose ASD based
behavioral criteria like repetitive behaviors. That means that it’s hard to discuss the idea of a “cure”
for autism, but it may be possible, using CRISPR-Gold, to treat the fragile X syndrome that causes
ASD-correlated behaviors in some individuals.
They injected specific parts of each mouse’s brain with gold nanoparticles treated to carry the Cas9
molecules. The CRISPR-Gold carried the Cas9 to a receptor called metabotropic glutamate receptor
5. Although this is a common receptor, in the part of the brain that the researchers injected it’s
related to repetitive behaviors. There in the striatum, the Cas9 shut down the gene that produces the
receptor. The amount of repetitive digging and leaping the mice were doing went down—in the case
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CRISPR could use gold nanoparticles to edit your brain | Popular Science https://www.popsci.com/crispr-gold-autism-spectrum#page-3
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2 of 3 6/26/2018, 2:06 AM
CRISPR could use gold nanoparticles to edit your brain | Popular Science https://www.popsci.com/crispr-gold-autism-spectrum#page-3
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