Gene-Editing Tool CRISPR Opens Door to New Autism Treatments

Source: Gene-Editing Tool CRISPR Opens Door to New Autism Treatments

A simple nip and tuck of the genetic code could erase autism for good, according to a new study.

Using gene-editing tool CRISPR, scientists were able to eliminate traits associated with the developmental disorder—in mice.

The genetically altered critters are the first step toward a promising future: Once offending DNA was edited out, scientists saw a 30 percent drop in compulsive digging and a 70 percent reduction in jumping, Newsweek reported, citing a study published by the journal Nature Biomedical Engineering.

Using a thin needle to inject CRISPR-Cas9 into the brain tissue, researchers were able to target the striatum—a region known to mediate habit formation.

Post-procedure, the recovered rodents seemed to morph into more calm, collected animals.

“You could knock out disease-causing genes and actually see fairly significant behavioral changes,” bioengineer Niren Murthy, inventor of the CRISPR-Gold technique administered to the mice, told Newsweek.

It’s unclear when (or even if) this technology will be ready for human testing. But in the meantime, this mouse measure should encourage people struggling with autism.

“I really want to give hope for patients and families,” lead study author Hye Young Lee, an assistant professor at the University of Texas Health Science Center at San Antonio, told Newsweek. “We are working on it, and they should not lose hope.”

Autism is a developmental disorder characterized by troubles with social interaction and communication, as well as restricted and repetitive behavior, like rocking, flapping limbs, or spinning (or, in the case of mice, obsessively burrowing).

About one in 59 children fall somewhere on the autism spectrum, which encompasses a wide variety of conditions caused by genetic and environmental influencers—the former of which could be corrected with CRISPR.

“It is such a social burden,” Lee said. “We want to help them.”

Moving forward, scientists hope to test the gene-editing technique on larger animals, like rats and monkeys, and eventually, humans.

“I am optimistic about the future,” Lee added.

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Published at Thu, 28 Jun 2018 17:08:49 +0000