Focused ultrasound-mediated brain genome editing

Proc Natl Acad Sci U S A. 2023 Aug 22;120(34):e2302910120. doi: 10.1073/pnas.2302910120. Epub 2023 Aug 14.


Gene editing in the brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches mainly rely on local injection to bypass the BBB. However, such administration is highly invasive and not amenable to treating certain delicate regions of the brain. We demonstrate a safe and effective gene editing technique by using focused ultrasound (FUS) to transiently open the BBB for the transport of intravenously delivered CRISPR/Cas9 machinery to the brain.

Keywords: CRISPR/Cas9; blood–brain barrier; focused ultrasound; gene delivery; genome editing.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biological Transport
  • Blood-Brain Barrier
  • Brain* / diagnostic imaging
  • Gene Editing*
  • Microbubbles