Focused ultrasound-mediated brain genome editing

Y.-H. Lao; R. Ji; J. K. Zhou; K. J. Snow; N. Kwon; E. Saville; S. He; S. Chauhan; C.-W. Chi; M. S. Datta; H. Zhang; C. H. Quek; S. Cai; M. Li; Y. Gaitan; L. Bechtel; S.-Y. Wu; C. M. Lutz; R. Tomer; S. A. Murray; A. Chavez; E. E. Konofagou; K. W. Leong

doi:10.1073/pnas.2302910120

BioengineeringbioRxivDec 10, 2022

Focused ultrasound-mediated brain genome editing

Y.-H. Lao, R. Ji, J. K. Zhou, K. J. Snow, N. Kwon, E. Saville, S. He, S. Chauhan, C.-W. Chi, M. S. Datta, H. Zhang, C. H. Quek, S. Cai, M. Li, Y. Gaitan, L. Bechtel, S.-Y. Wu, C. M. Lutz, R. Tomer, S. A. Murray, A. Chavez, E. E. Konofagou, K. W. Leong

Abstract

Gene editing in the mammalian brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches 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.

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