Brief intro:
- Author: 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
- Journal: Res Sq
- Doi: https://www.doi.org/10.21203/rs.3.rs-2365576/v1
- Publication Date: 2023 Jan 20
Products/Services used in the paper
Quotation shows PackGene: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).
Research Field:gene editing in brain
AAV Serotype:AAV9
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.
About PackGene
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