Yeh-Hsing Lao, Robin Ji, Joyce K. Zhou, Kathy J. Snow, Nancy Kwon, Ethan Saville, Siyu He, Shradha Chauhan, Chun-Wei Chi, Malika S. Datta, Hairong Zhang, Chai Hoon Quek, S. Sarah Cai, Mingqiang Li, Yaned Gaitan, Lawrence Bechtel, Shih-Ying Wu, Cathleen M. Lutz, Raju Tomer, Stephen A. Murray, Alejandro Chavez, Elisa E. Konofagou
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.
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