Leong Kam, Lao Yeh-Hsing, Ji Robin, Zhou Joyce, Snow Kathy, Kwon Nancy, Saville Ethan, He Siyu, Chauhan Shradha, Chi Chun-Wei, Datta Malika, Zhang Hairong, Quek Chai Hoon, Cai Sarah, Li Mingqiang, Gaitan Yaned, Bechtel Lawrence, Wu Shih-Ying, Lutz Cathleen, Tomer Raju, Murray Stephen, Chavez Alejandro, Konofagou Elisa
Columbia University.
University at Buffalo.
Res Sq. 2023 Jan 20:rs.3.rs-2365576. doi: 10.21203/rs.3.rs-2365576/v1.
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.
由于血脑屏障（BBB）所施加的受限运输，在哺乳动物大脑中进行基因编辑一直具有挑战性。当前的方法依赖于局部注射以绕过血脑屏障。然而，这种给药方式具有高度侵入性，并且不适用于治疗大脑的某些精细区域。我们通过使用聚焦超声（FUS）来短暂打开血脑屏障，以便将静脉注射的CRISPR/Cas9机制运输到大脑，从而证明了一种安全有效的基因编辑技术。
