Focused ultrasound-mediated brain genome editing

Res Sq. 2023 Jan 20
Kam Leong, Yeh-Hsing Lao, Robin Ji, Joyce Zhou, Kathy Snow, Nancy Kwon, Ethan Saville, Siyu He, Shradha Chauhan, Chun-Wei Chi, Malika Datta, Hairong Zhang, Chai Hoon Quek, Sarah Cai, Mingqiang Li, Yaned Gaitan, Lawrence Bechtel, Shih-Ying Wu, Cathleen Lutz, Raju Tomer, Stephen Murray, Alejandro Chavez, and Elisa Konofagou

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AAV vector packaging	The AAVs used in this study were either produced in-house (for the experiments done in C57BL/6 mice) or by PackGene (for the validations done in Ai9 and TLR2 mice).	Request Quote

Research Field: gene editing in brain

AAV Serotype: AAV9

Dose: 2×10^12 GC/mouse

Routes of Administration: Adult Ai9 mice received our Ai9- targeting vector intravenously under the FUS array system.

Targeted organ: brain

Animal or cell line strain: The AAVs used in this study were either produced in-house (for the experiments done in C57BL/6 mice) or by PackGene (for the validations done in Ai9 and TLR2 mice).

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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