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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Veröffentlicht in:Proceedings of the National Academy of Sciences - PNAS 2023-08, Vol.120 (34), p.e2302910120-e2302910120
Hauptverfasser: 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: Artikel
Sprache:eng
Schlagworte:
Biological Sciences
Biological Transport
Blood-Brain Barrier
Brain - diagnostic imaging
CRISPR
Early-Career-Researcher
Editing
Gene Editing
Genetic modification
Genomes
Microbubbles
Ultrasonic testing
Ultrasound
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Beschreibung
Zusammenfassung: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.
ISSN:0027-8424
1091-6490
DOI:10.1073/PNAS.2302910120