Department of Biomedical Engineering, Columbia University, New York, NY 10027.
Department of Pharmaceutical Sciences, University at Buffalo, The State University of New York, Buffalo, NY 14214.
Proc Natl Acad Sci U S A. 2023 Aug 22;120(34):e2302910120. doi: 10.1073/pnas.2302910120. Epub 2023 Aug 14.
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.
脑内基因编辑一直具有挑战性，原因是血脑屏障（BBB）限制了物质的运输。目前的方法主要依赖于局部注射来绕过 BBB。然而，这种给药方式具有很强的侵入性，并且不适合治疗大脑的某些精细区域。我们通过使用聚焦超声（FUS）短暂打开 BBB，使经静脉递送至大脑的 CRISPR/Cas9 机器能够运输，从而展示了一种安全有效的基因编辑技术。
