摘要
Gene therapy,as one of the popular therapies nowadays,has been applied with great success in various diseases.Nevertheless,there are also concerns about the safety,therapeutic efficacy,and applicability of gene therapy.Microbubble-assisted ultrasound-guided gene therapy offers the following incomparable advantages over conventional methods.Initially,this strategy enables the therapeutic nucleic acids to effectively cross the barriers like cell membrane,blood-brain barrier,and blood-retinal barrier that exist in the body and reach regions that cannot be accessed by conventional methods,which extends the scope of applications of gene therapy.Next,it is possible to restrict the zone of gene transfection to the lesion area by controlling the irradiation coverage of ultrasound,hence effectively controlling the risk of off-targeting.Additionally,with the support of various imaging techniques like ultrasound imaging and magnetic resonance imaging,the procedure of gene therapy can be under image guidance,which will dramatically improve the safety of gene therapy.This review will highlight on the advances in ultrasound-enhanced gene delivery strategy,attention to the relevant mechanisms of the strategy,the derived technologies and the related cutting-edge applications.Moreover,we will also have a discussion on the limitations and future prospects associated with this gene delivery strategy.
基因治疗作为当今流行的疗法之一,在各种疾病的治疗中取得了巨大的成功.然而,基因治疗的安全性、疗效和适用性也备受关注.微泡辅助超声引导下的基因治疗与传统方法相比具有以下优势:首先,这种策略能使治疗核酸有效地通过体内存在的天然屏障,如细胞膜屏障、血脑屏障、血视网膜屏障,可使药物富集在传统递送方法无法覆盖的病灶区域,进一步扩大了基因治疗的应用范围.其次,可以通过控制超声波的照射范围,将基因转染区域限制在病变区域,从而有效控制基因治疗脱靶的风险.此外,在超声成像和磁共振成像等多种成像技术的支持下,基因治疗过程可以在图像引导下进行,这将大大提高基因治疗的安全性.本综述重点介绍了超声波增强基因递送策略的进展,关注该策略的相关机制、衍生技术和相关前沿应用,并对该基因递送策略的局限性和未来前景进行了讨论.
作者
Zhen Tie
Shuhao Zhang
Yunfeng Qu
Mengxuan Wang
Renfa Liu
Desheng Sun
Zhifei Dai
铁真;张书豪;屈云凤;王梦玄;刘仁发;孙德胜;戴志飞(Department of Biomedical Engineering,College of Future Technology,National Biomedical Imaging Center,State Key Laboratory of Vascular Homeostasis and Remodeling,Peking University,Beijing 100871,China;Department of Ultrasonic Imaging,Peking University Shenzhen Hospital,Shenzhen 518035,China)
基金
financially supported by the National Natural Science Foundation of China(81930047 to Dai Z,82102062 to Liu R)
Sanming Project of Medicine in Shenzhen(SZSM202111011)。
