Brain-targeted CRISPR could treat autism behaviors

Researchers at University of Texas Health Science Center, University of California Berkeley and GenEdit Inc. (Berkeley, Calif.) used gold nanoparticles as a non-viral method to deliver the CRISPR gene editing complex into the brain and showed that delivery of CRISPR-Cas9 using the nanoparticles could treat symptoms of Fragile X syndrome.

Current methods to deliver CRISPR-Cas9 (CRISPR-associated protein 9) and single guide RNA (sgRNA) usually involve viral administration. However, these can lead to immunogenicity of the viral vectors or off-target genomic damage due to prolonged expression. In the brain, this could lead to changes in neuronal phenotypes, according to the authors in a paper published in Nature Biomedical Engineering...