Document Type
Article
Publication Date
8-22-2023
Original Citation
Lao Y,
Ji R,
Zhou J,
Snow KJ,
Kwon N,
Saville E,
He S,
Chauhan S,
Chi C,
Datta M,
Zhang H,
Quek C,
Cai S,
Li M,
Gaitan Y,
Bechtel L,
Wu S,
Lutz C,
Tomer R,
Murray SA,
Chavez A,
Konofagou E,
Leong K.
Focused ultrasound-mediated brain genome editing. Proc Natl Acad Sci U S A. 2023;120(34):e2302910120.
Keywords
JMG, Gene Editing, Brain, Blood-Brain Barrier, Biological Transport, Microbubbles
JAX Source
Proc Natl Acad Sci U S A. 2023;120(34):e2302910120.
ISSN
1091-6490
PMID
37579143
DOI
https://doi.org/10.1073/pnas.2302910120
Grant
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.).
Abstract
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.
Comments
Copyright © 2023 the Author(s). Published by PNAS. This open access article is distributed under Creative Commons AttributionNonCommercialNoDerivatives License 4.0 (CC BYNCND).