高分子基因载体的最新进展:传递机制、分子设计及应用。

IF 2.9 Q2 BIOPHYSICS
Biophysics reviews Pub Date : 2023-03-01 Epub Date: 2023-03-28 DOI:10.1063/5.0123664
Kemao Xiu, Jifeng Zhang, Jie Xu, Y Eugene Chen, Peter X Ma
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引用次数: 0

摘要

近年来,基因治疗和基因传递受到了广泛关注,特别是为预防冠状病毒引起的严重症状而开发的COVID-19 mRNA疫苗。将DNA和RNA等基因送入细胞,是基因治疗成功的关键一步,也是一个瓶颈。为了解决这个问题,可以将基因装载和传递到细胞中的载体(载体)被开发出来,包括病毒载体和非病毒载体。尽管病毒基因载体具有相当的转染效率,并且自COVID-19疫苗应用以来,基于脂质的基因载体得到了广泛应用,但其潜在的免疫和生物安全性问题限制了其应用。另外,与基于病毒和脂质的载体相比,聚合基因载体更安全、更便宜、更通用。近年来,各种分子设计良好的聚合基因载体被开发出来,它们要么具有较高的转染效率,要么具有一定的应用优势。本文综述了近年来高分子基因载体的研究进展,包括转染机制、分子设计和生物医学应用。还介绍了市售的聚合基因载体/试剂。该领域的研究人员从未停止过通过合理的分子设计和生物医学评估来寻求安全高效的聚合基因载体。近年来的研究成果大大加快了聚合基因载体的临床应用进程。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Recent progress in polymeric gene vectors: Delivery mechanisms, molecular designs, and applications.

Gene therapy and gene delivery have drawn extensive attention in recent years especially when the COVID-19 mRNA vaccines were developed to prevent severe symptoms caused by the corona virus. Delivering genes, such as DNA and RNA into cells, is the crucial step for successful gene therapy and remains a bottleneck. To address this issue, vehicles (vectors) that can load and deliver genes into cells are developed, including viral and non-viral vectors. Although viral gene vectors have considerable transfection efficiency and lipid-based gene vectors become popular since the application of COVID-19 vaccines, their potential issues including immunologic and biological safety concerns limited their applications. Alternatively, polymeric gene vectors are safer, cheaper, and more versatile compared to viral and lipid-based vectors. In recent years, various polymeric gene vectors with well-designed molecules were developed, achieving either high transfection efficiency or showing advantages in certain applications. In this review, we summarize the recent progress in polymeric gene vectors including the transfection mechanisms, molecular designs, and biomedical applications. Commercially available polymeric gene vectors/reagents are also introduced. Researchers in this field have never stopped seeking safe and efficient polymeric gene vectors via rational molecular designs and biomedical evaluations. The achievements in recent years have significantly accelerated the progress of polymeric gene vectors toward clinical applications.

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CiteScore
3.60
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