Focused ultrasound-mediated brain genome editing.
Yeh-Hsing LaoRobin JiJoyce K ZhouKathy J SnowNancy KwonEthan SavilleSiyu HeShradha ChauhanChun-Wei ChiMalika S DattaHairong ZhangChai Hoon QuekS Sarah CaiMingqiang LiYaned GaitanLawrence BechtelShih-Ying WuCathleen M LutzRaju TomerStephen A MurrayAlejandro ChavezElisa E KonofagouKam W LeongPublished in: Proceedings of the National Academy of Sciences of the United States of America (2023)
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.