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Stanford Researchers Achieve Breakthrough in Precision RNA Delivery for Neuronal Repair

CRISPR-TO technology demonstrates 50% neurite growth in vitro and launches large-scale screening for neurodegenerative therapies.

Typically, CRISPR is used to slice and edit genetic code, but in this case the researchers didn’t want to make any changes. Credit: Neuroscience News

Overview

  • CRISPR-TO, an engineered CRISPR-Cas13 system, delivers RNA to precise subcellular locations, enabling targeted neuronal repair.
  • In vitro experiments on mouse neurons showed RNA delivery to neurite tips, with one RNA promoting up to 50% neurite growth within 24 hours.
  • The technology represents a paradigm shift from DNA editing to spatially precise RNA transport, forming the foundation of 'spatial RNA medicine.'
  • Researchers are now screening additional RNA molecules in mouse and human neurons to identify candidates for treating ALS, spinal muscular atrophy, and spinal cord injuries.
  • The findings, published in *Nature* on May 21, 2025, highlight the potential for CRISPR-TO to revolutionize therapies for neurodegenerative diseases and injuries.