A review of the tortuous path of nonviral gene delivery and recent progress.
A review of the tortuous path of nonviral gene delivery and recent progress.
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DOI：
10.1016/j.ijbiomac.2021.05.192
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发表时间：
2021-07-31
影响因子：
8.2
通讯作者：
Layek B
中科院分区：
文献类型：
--
作者：
Sharma D;Arora S;Singh J;Layek B
基因治疗包括将外源遗传物质转移到患者的靶细胞中以治疗或预防疾病。然而，将遗传物质转移到所需细胞中是具有挑战性的，并且通常需要专门的工具或递送系统。在过去的40年里，科学家们主要是追求各种病毒作为基因传递载体，总体进展缓慢，与预期相去甚远。作为替代方案，非病毒载体由于其几个优点而获得了大量关注，包括上级安全特性、增强的有效载荷能力和隐形能力。由于非病毒载体遇到多个细胞外和细胞内的障碍，限制了转移到靶细胞核的遗传负载，我们已经详细讨论了这些障碍在这篇评论。利用物理方法如电穿孔、声致穿孔、基因枪的直接方法消除了对用于基因递送的特定载体的需要。相比之下，基因转移的化学方法利用天然或合成化合物作为载体，以增加细胞靶向和基因治疗的有效性。我们还强调了最近的进展，以加强目前的非病毒方法。因此，在这篇综述中，我们重点讨论了目前正在发展的非病毒基因递送系统及其未来的前景。
Gene therapy encompasses the transfer of exogenous genetic materials into the patient’s target cells to treat or prevent diseases. Nevertheless, the transfer of genetic material into desired cells is challenging and often requires specialized tools or delivery systems. For the past 40 years, scientists are mainly pursuing various viruses as gene delivery vectors, and the overall progress has been slow and far from the expectation. As an alternative, nonviral vectors have gained substantial attention due to their several advantages, including superior safety profile, enhanced payload capacity, and stealth abilities. Since nonviral vectors encounter multiple extra- and intra-cellular barriers limiting the transfer of genetic payload into the target cell nucleus, we have discussed these barriers in detail in this review. A direct approach, utilizing physical methods like electroporation, sonoporation, gene gun, eliminate the requirement for a specific carrier for gene delivery. In contrast, chemical methods of gene transfer exploit natural or synthetic compounds as carriers to increase cellular targeting and gene therapy effectiveness. We have also emphasized the recent advancements toward enhancing the current nonviral approaches. Therefore, in this review, we have focused on discussing the current evolving nonviral gene delivery systems and their future perspectives.
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影响因子：
5.1
作者：
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通讯作者：
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