Gene therapy has been widely investigated for the treatment of genetic, acquired, and infectious diseases. Pioneering work utilized viral vectors; however, these are suspected of causing serious adverse events, resulting in the termination of several clinical trials. Non-viral vectors, such as lipid nanoparticles, have attracted significant interest, mainly due to their successful use in vaccines in the current COVID-19 pandemic. Although they allow safe delivery, they come with the disadvantage of off-target delivery. The application of ultrasound to ultrasound-sensitive particles allows for a direct, site-specific transfer of genetic materials into the organ/site of interest. This process, termed ultrasound-targeted gene delivery (UTGD), also increases cell membrane permeability and enhances gene uptake. This review focuses on the advances in ultrasound and the development of ultrasonic particles for UTGD across a range of diseases. Furthermore, we discuss the limitations and future perspectives of UTGD.

基因疗法已被广泛研究用于治疗遗传性疾病、获得性疾病和传染性疾病。开创性工作利用了病毒载体；然而，这些被怀疑会导致严重的不良事件，导致多项临床试验终止。非病毒载体，如脂质纳米颗粒，引起了人们的极大兴趣，这主要是由于它们在当前 COVID-19 大流行中成功用于疫苗。尽管它们允许安全递送，但它们具有脱靶递送的缺点。超声对超声敏感颗粒的应用允许将遗传物质直接、特定于位点转移到感兴趣的器官/位点。这个过程，称为超声靶向基因传递 (UTGD)，也增加细胞膜通透性并增强基因摄取。本综述侧重于超声的进展和用于 UTGD 的超声粒子在一系列疾病中的发展。此外，我们还讨论了 UTGD 的局限性和未来前景。