Department of Nanobiotechnology, Faculty of Biological Sciences, Tarbiat Modares University, Tehran, Iran.
Monoclonal Antibody Research Center, Avicenna Research Institute, ACECR, Tehran, Iran.
Protein Pept Lett. 2020;27(8):698-710. doi: 10.2174/0929866527666200206153328.
Gene-based therapy largely relies on the vector type that allows a selective and efficient transfection into the target cells with maximum efficacy and minimal toxicity. Although, genes delivered utilizing modified viruses transfect efficiently and precisely, these vectors can cause severe immunological responses and are potentially carcinogenic. A promising method of overcoming this limitation is the use of non-viral vectors, including cationic lipids, polymers, dendrimers, and peptides, which offer potential routes for compacting DNA for targeted delivery. Although non-viral vectors exhibit reduced transfection efficiency compared to their viral counterpart, their superior biocompatibility, non-immunogenicity and potential for large-scale production make them increasingly attractive for modern therapy. There has been a great deal of interest in the development of biomimetic chimeric peptides. Biomimetic chimeric peptides contain different motifs for gene translocation into the nucleus of the desired cells. They have motifs for gene targeting into the desired cell, condense DNA into nanosize particles, translocate the gene into the nucleus and enhance the release of the particle into the cytoplasm. These carriers were developed in recent years. This review highlights the stepwise development of the biomimetic chimeric peptides currently being used in gene delivery.
基因治疗在很大程度上依赖于载体类型，这种载体类型允许选择性和高效地转染到靶细胞中，同时最大限度地提高疗效和最小化毒性。虽然利用修饰病毒递送的基因可以有效地进行转染，但是这些载体可能会引起严重的免疫反应，并且具有潜在的致癌性。克服这一限制的一种有前途的方法是使用非病毒载体，包括阳离子脂质体、聚合物、树枝状聚合物和肽，它们为靶向递送的 DNA 提供了潜在的压缩途径。虽然非病毒载体的转染效率比其病毒对应物低，但它们优越的生物相容性、非免疫原性和大规模生产的潜力使它们在现代治疗中越来越有吸引力。人们对仿生嵌合肽的开发产生了浓厚的兴趣。仿生嵌合肽包含不同的基序，用于将基因转染到所需细胞的核内。它们具有将基因靶向到所需细胞的基序，将 DNA 浓缩成纳米大小的颗粒，将基因转染到核内，并增强颗粒向细胞质的释放。这些载体是近年来开发的。本综述重点介绍了目前用于基因传递的仿生嵌合肽的逐步发展。
