Researchers are making rapid progress in using CRISPR-based gene editing to treat neurological disorders, with recent mouse experiments delivering remarkable results. These advances could bring human trials just a few years away.

Until now, the brain has been one of the hardest organs to target due to the blood–brain barrier. New delivery methods are being explored to safely transport gene-editing tools into brain cells.

In one major study, scientists used prime editing to repair a mutation causing alternating hemiplegia of childhood (AHC), improving cognition, motor skills, and lifespan in mice. Other teams are working on treatments for Huntington’s disease, Friedreich’s ataxia, and MEF2C-related disorders.

Although these therapies are still in preclinical stages, experts say the data suggest that editing the brain’s DNA is “less science fiction, and closer to reality.”

Source: Nature