Company GenEdit, founded by Berkeley PhDs Kunwoo Lee and Hyo Min Park, with Berkeley bioengineering professor Niren Murthy, was part of a study representing the first demonstration of behavior change of a living animal through non-viral gene editing.
Berkeley News: 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.
“There are no treatments or cures for autism yet, and many of the clinical trials of small-molecule treatments targeting proteins that cause autism have failed,” said study leader Hye Young Lee, an assistant professor of cellular and integrative physiology at the University of Texas Health Science Center at San Antonio. “This is the first case where we were able to edit a causal gene for autism in the brain and show rescue of the behavioral symptoms.”