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Stanford scientists have developed a groundbreaking CRISPR-based technology that can deliver RNA to damaged parts of neurons, offering hope for future treatments of neurodegenerative diseases like ALS and spinal muscular atrophy. Published in Nature on May 21, the study was led by postdoctoral researcher Mengting Han and senior author Professor Stanley Qi of Stanford University.
Normally, when neurons are injured, RNA molecules help repair them by creating essential proteins. But in neurological disorders or spinal cord injuries, the cellular machinery that moves RNA to the right locations breaks down, preventing healing. The new technique, called CRISPR-TO (Targeted Organization), uses a modified version of the CRISPR-Cas13 system—not to edit RNA, but to transport it like a molecular “mailman” to specific spots in the neuron using localization signals or "zip codes."
When tested in mouse brain cells, CRISPR-TO successfully delivered RNA to the tips of neurites—the structures neurons use to connect with each other—boosting regrowth by up to 50% in just one day. The team is now screening other RNA molecules that could further enhance neuron repair, potentially laying the groundwork for a new field of “spatial RNA medicine.”
This innovation could lead to safer and more effective RNA-based therapies by ensuring that molecules are not only present in cells but precisely located where they’re most needed. The research was supported by multiple institutions, including the NIH and the American Heart Association.
