Focused ultrasound-mediated brain genome editing

Focused ultrasound-mediated brain genome editing

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 t...

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Bibliographic Details
Published in:Proceedings of the National Academy of Sciences - PNAS Vol. 120; no. 34; p. e2302910120
Main Authors: Lao, Yeh-Hsing, Ji, Robin, Zhou, Joyce K, Snow, Kathy J, Kwon, Nancy, Saville, Ethan, He, Siyu, Chauhan, Shradha, Chi, Chun-Wei, Datta, Malika S, Zhang, Hairong, Quek, Chai Hoon, Cai, S Sarah, Li, Mingqiang, Gaitan, Yaned, Bechtel, Lawrence, Wu, Shih-Ying, Lutz, Cathleen M, Tomer, Raju, Murray, Stephen A, Chavez, Alejandro, Konofagou, Elisa E, Leong, Kam W
Format: Journal Article
Language:English
Published: United States National Academy of Sciences 22-08-2023
Series:Brief Report
Subjects:
Biological Sciences
Biological Transport
Blood-Brain Barrier
Brain - diagnostic imaging
CRISPR
Early-Career-Researcher
Editing
Gene Editing
Genetic modification
Genomes
Microbubbles
Ultrasonic testing
Ultrasound
gene delivery
genome editing
blood–brain barrier
CRISPR/Cas9
focused ultrasound
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Summary: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.
Bibliography:ObjectType-Article-1
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Edited by Jeremy Nathans, Johns Hopkins University School of Medicine, Baltimore, MD; received March 7, 2023; accepted June 28, 2023
1Y.-H.L. and R.J. contributed equally to this work.
ISSN:0027-8424
1091-6490
DOI:10.1073/PNAS.2302910120