Treating Autism-Related Behaviors Using CRISPR Gold Nanoparticles

Used for gene editing and considered one of the century’s most important discoveries, CRISPR/CAS9 genetic technologycontinues to see new developments every day. In June 2018, a study published in Natureused CRISPR and gold nanoparticles in mice to successfully correct the gene causing Fragile-X syndrome, considered within autism spectrum disorder.

Researchers at the University of California, United States, aimed to edit genes in mice with Fragile-X syndrome using CRISPR-gold nanoparticles. Fragile-X is a genetic disease associated with autism spectrum disorder. One important finding is involuntary repeated motor behavior.

Gold nanoparticles carrying the CRISPR system were injected into mouse brains. The targeted gene was thought responsible for repetitive behaviors , metabotropic glutamate receptor 5.

Following injection, repetitive behaviors in mice decreased by up to 70 percent.

Researchers also successfully edited genes in different brain cells using CRISPR-gold nanoparticles.

So What Does This Study Mean for Us?

• This study shows for the first time that gene editing in the brain with CRISPR can be effective without using a virus . This approach could help treat people with neurological diseases such as Hungtington and epilepsy.
• Gene editing with viruses was a very dangerous method that could have side effects. Using CRISPR-gold nanoparticles, however, gene editing can be performed without any side effects.
• It should be emphasized that this method is not a complete cure for autism. Gene editing in individuals with autism spectrum disorder is also an ethical issue for debate. However, this method may reduce symptoms such as repetitive behaviors in autistic individuals. This was a laboratory study in mice and needs support from further studies and clinical trials.

What is AUTISM? Vaccines, Special Diets and Its Relationship with Cancer

In the video below, mice showing autistic behavior through obsessive digging are shown to move less compulsively after the problematic gene is removed with CRISPR.