Proc Natl Acad Sci U S A. 2023 Aug 22
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
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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: genome editing in brain

Keywords: genome editing, CRISPR/Cas9, blood–brain barrier, focused ultrasound, gene delivery

AAV Serotype: AAV9

Dose: Adult female and male reporter mice (Ai9 or TLR2; 9-10 weeks old; JAX Lab) were given with 2×10^12 vg/mouse AAV9 encoding SaCas9 and reporter-specific gRNAs under the FUS array treatment.

Routes of Administration: FUS array treatment

Targeted organ: brain

Animal or cell line strain: mice (Ai9 or TLR2; 9-10 weeks old; JAX Lab)


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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