Applications of focused ultrasound-mediated blood-brain barrier opening.

The blood brain barrier (BBB) plays a critically important role in the regulation of central nervous system (CNS) homeostasis, but also represents a major limitation to treatments of brain pathologies. In recent years, focused ultrasound (FUS) in conjunction with gas-filled microbubble contrast agents has emerged as a powerful tool for transiently and non-invasively disrupting the BBB in a targeted and image-guided manner, allowing for localized delivery of drugs, genes, or other therapeutic agents. Beyond the delivery of known therapeutics, FUS-mediated BBB opening also demonstrates the potential for use in neuromodulation and the stimulation of a range of cell- and tissue-level physiological responses that may prove beneficial in disease contexts. Clinical trials investigating the safety and efficacy of FUS-mediated BBB opening are well underway, and offer promising non-surgical approaches to treatment of devastating pathologies. This article reviews a range of pre-clinical and clinical studies demonstrating the tremendous potential of FUS to fundamentally change the paradigm of treatment for CNS diseases.

血脑屏障（BBB）在调节中枢神经系统（CNS）内稳态方面起着至关重要的作用，但也对脑部疾病的治疗构成了重大限制。近年来，聚焦超声（FUS）结合充气微泡造影剂已成为一种强大的工具，能够以靶向和影像引导的方式暂时且非侵入性地破坏血脑屏障，从而实现药物、基因或其他治疗剂的局部递送。除了递送已知的治疗药物外，聚焦超声介导的血脑屏障开放还显示出在神经调节以及刺激一系列细胞和组织水平生理反应方面的潜力，这些在疾病背景下可能是有益的。探究聚焦超声介导的血脑屏障开放的安全性和有效性的临床试验正在顺利进行，并为治疗毁灭性疾病提供了有前景的非手术方法。本文综述了一系列临床前和临床研究，这些研究表明聚焦超声在从根本上改变中枢神经系统疾病治疗模式方面具有巨大潜力。