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精准医学聚焦于中枢神经系统:应用聚焦超声和微泡的无创治疗药物输送。

Precision medicine focus on the central nervous system: Non-invasive therapeutic agent delivery with focused ultrasound and microbubbles.

机构信息

Department of Human and Molecular Genetics, Virginia Commonwealth University, School of Medicine, Richmond, VA, United States.

VCU Institute of Molecular Medicine, Virginia Commonwealth University, School of Medicine, Richmond, VA, United States.

出版信息

Adv Cancer Res. 2024;164:191-240. doi: 10.1016/bs.acr.2024.06.003. Epub 2024 Jul 2.

DOI:10.1016/bs.acr.2024.06.003
PMID:39306366
Abstract

Focused ultrasound (FUS) combined with microbubble (MB) treatment is a promising strategy capable of accurately delivering molecular medicines and gene therapy to treat various disease states. The rapid progression and use of FUS technology, from its inception to applications in contemporary medicine, exemplifies the significance and expanding potential of this technology. FUS for drug delivery in the brain can overcome challenging obstacles posed by the blood-brain barrier (BBB) in treating central nervous system (CNS) disorders. Both FUS and magnetic resonance imaging-guided FUS are non-invasive techniques for effectively opening the BBB and enhancing the transportation of molecular medicines and imaging agents into the brain. By integrating MBs into this process, it is possible to disrupt the BBB, facilitating delivery of therapeutic compounds including neuropeptides, proteins, antibodies, chemotherapeutic drugs and recently viruses accurately into the CNS. The safety and versatility of ultrasound makes it an attractive approach for administering molecular medicines, with potential applications extending beyond neurological disorders to include cancer treatment and other medical fields. Preclinical and clinical studies confirm that FUS is safe and efficient in enhancing drug administration, particularly where delivery to a precise location in the CNS is required. Combination therapies that utilize FUS and MBs also provide synergistic responses in cancer therapy. Further refining FUS and MB approaches both from a mechanical and reagent perspective will be forthcoming in the future and prove valuable in precisely defining targets and broadening therapeutic applications. Continued development and applications of FUS and MB technologies will improve therapeutic outcomes and advance patient care in multiple diseases states. This will elevate FUS and MBs from infrequently used medical options to mainstream medical applications.

摘要

聚焦超声(FUS)联合微泡(MB)治疗是一种很有前途的策略,能够将分子药物和基因治疗精确递送到各种疾病状态。FUS 技术的快速发展和应用,从其起源到当代医学的应用,都体现了这项技术的重要性和不断扩大的潜力。FUS 用于脑部药物输送,可以克服血脑屏障(BBB)在治疗中枢神经系统(CNS)疾病方面的挑战。FUS 和磁共振成像引导的 FUS 都是非侵入性技术,可以有效地打开 BBB,并增强分子药物和成像剂向大脑的输送。通过将 MB 整合到这个过程中,可以破坏 BBB,促进包括神经肽、蛋白质、抗体、化疗药物和最近的病毒在内的治疗化合物准确递送到 CNS。超声的安全性和多功能性使其成为一种有吸引力的分子药物给药方法,其潜在应用不仅限于神经疾病,还包括癌症治疗和其他医学领域。临床前和临床研究证实,FUS 在增强药物给药方面是安全有效的,特别是在需要将药物精确递送到 CNS 的特定位置时。FUS 和 MB 联合的组合疗法在癌症治疗中也提供协同反应。从机械和试剂的角度进一步改进 FUS 和 MB 方法将在未来出现,并在精确定义靶点和拓宽治疗应用方面证明是有价值的。FUS 和 MB 技术的不断发展和应用将改善多种疾病状态的治疗效果并改善患者护理。这将使 FUS 和 MB 从很少使用的医疗选择提升为主流的医疗应用。

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