Research towards a treatment for autism

Mice with a form of autism causing them to dig obsessively, dug less obsessively when researchers knocked out a gene involved in repetitive behavior.

Video by Roxanne Makasdjian and Stephen McNally, with video from Hye Young Lee, assistant professor of cellular and integrative physiology at the University of Texas Health Science Center at San Antonio.

Video written & edited by Roxanne Makasdjian & Steve McNally

Berkeley — Scientists have used CRISPR-Cas9 gene editing to lessen some autism symptoms in mice with a form of fragile X syndrome, the most common known single-gene cause of autism spectrum disorder.

Employing gold nanoparticles to deliver the DNA-cutting Cas9 enzyme into the brain – a technique developed at the University of California, Berkeley, and called CRISPR-Gold – the researchers were able to edit the gene for a neurotransmitter receptor and reduce the repetitive behavior characteristic of fragile X syndrome (FXS).

Because exaggerated repetitive behaviors are common features in autism spectrum disorders, the efficient reduction of these behaviors in FXS mouse models demonstrates the potential application of this technique to other types of autism for which the genetic cause is known, the researchers say.

“The really compelling thing about this paper is that Hye Young was able to show that if you injected CRISPR-Gold into the brain, you could knock out disease-causing genes and actually see fairly significant behavioral changes,” said CRISPR-Gold inventor Niren Murthy, a UC Berkeley professor of bioengineering. “This is the first time anyone had ever shown that with non-viral delivery.”
Cont'd... For full story: http://news.berkeley.edu/2018/06/25/crispr-reduces-autism-symptoms-in-mice/

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