Focused ultrasound-mediated brain genome editing. Proc Natl Acad Sci U S A. 2023;120(34):e2302910120.
JMG, Gene Editing, Brain, Blood-Brain Barrier, Biological Transport, Microbubbles
Proc Natl Acad Sci U S A. 2023;120(34):e2302910120.
This work was supported by NIH SCGE Program (UG3NS115598, A.C., E.E.K., and K.W.L.; U42OD026635, C.M.L. and S.A.M.) and ASGCT Career Development Award (ASGCT2021003, Y.-H.L.).
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.