When a neuron in our body gets damaged, segments of RNA produce proteins that can help repair the injury. But in neurological disorders such as ALS and spinal muscular atrophy, or following spinal cord injuries, the mechanisms for moving life-essential RNA to injured sites within the cell fail. As a result, RNA molecules can't get to where they are needed and damage becomes permanent.
Researchers at Stanford have developed a technology for transporting RNA to specific locations within a neuron, where it can repair and even regrow parts of the cell. Their work, supported by the National Institutes of Health, forms the foundation for a new class of therapeutics that the researchers are calling "spatial RNA medicine," which they hope will lead to treatments for neurological diseases as well as traumatic injuries.
"For the first time, we've harnessed the power of CRISPR technology to create a precise spatial 'zip code' that delivers RNA molecules exactly where they're needed within cells," said Stanley Qi, an associate professor of bioengineering and senior author on the paper published May 21 in Nature. "Imagine being able to specifically target damaged sites within a neuron, repairing them, and promoting their regrowth - this is what our technology achieves."
A CRISPR-based mailman
In recent years, researchers have realized that the distribution of RNA within a cell - where specific molecules are located - may be just as important as what they are capable of doing. An individual neuron can be over a meter long, and aging, injury, and mutations can all disrupt its ability to transport the tiny RNA over such a distance.
"Therapeutic RNA can't help if it doesn't get to where it's needed," Qi said. "We wanted to create a technology that could reliably move RNA to where it needs to function."
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Imaging showing CRISPR-mediated long distance therapeutic RNA transport for repairing damage in a brain neuron, with the therapeutic RNA tagged red. | Mengting Han
