The Progress of Non-Viral Materials and Methods for Gene Delivery to Skeletal Muscle.
The Progress of Non-Viral Materials and Methods for Gene Delivery to Skeletal Muscle.
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DOI：
10.3390/pharmaceutics14112428
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发表时间：
2022-11-10
期刊：
影响因子：
5.4
通讯作者：
Wang G
中科院分区：
文献类型：
--
作者：
Cui Z;Jiao Y;Pu L;Tang JZ;Wang G
自从乔恩·A. Wolff发现骨骼肌细胞能够表达外源基因，Russell J. Mumper通过添加聚合物提高基因转染效率，骨骼肌已成为潜在的基因传递和表达靶点。已经开发了不同的方法来将转基因递送到骨骼肌中。其中，病毒载体可以实现有效的基因递送效率。然而，引发生物安全风险的可能性限制了其临床应用。因此，具有可靠生物相容性的非病毒生物材料介导的方法是肌肉内原位基因递送的有前途的工具。近年来，报道了一系列先进的非病毒基因传递材料及相关方法，如聚合物、脂质体、细胞穿透肽以及物理传递方法等。本文综述了非病毒肌内基因传递材料及相关方法的研究进展和面临的挑战，重点介绍了聚合物的研究成果和发展方向。
Since Jon A. Wolff found skeletal muscle cells being able to express foreign genes and Russell J. Mumper increased the gene transfection efficiency into the myocytes by adding polymers, skeletal muscles have become a potential gene delivery and expression target. Different methods have been developing to deliver transgene into skeletal muscles. Among them, viral vectors may achieve potent gene delivery efficiency. However, the potential for triggering biosafety risks limited their clinical applications. Therefore, non-viral biomaterial-mediated methods with reliable biocompatibility are promising tools for intramuscular gene delivery in situ. In recent years, a series of advanced non-viral gene delivery materials and related methods have been reported, such as polymers, liposomes, cell penetrating peptides, as well as physical delivery methods. In this review, we summarized the research progresses and challenges in non-viral intramuscular gene delivery materials and related methods, focusing on the achievements and future directions of polymers.
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