Suppr超能文献

基于生物材料的基因治疗。

Biomaterial-based gene therapy.

作者信息

Yu Yi, Gao Yijun, He Liming, Fang Bairong, Ge Wenhui, Yang Pu, Ju Yikun, Xie Xiaoyan, Lei Lanjie

机构信息

Department of Stomatology The Second Xiangya Hospital Central South University Changsha China.

Department of Stomatology Changsha Stomatological Hospital Changsha China.

出版信息

MedComm (2020). 2023 Jun 3;4(3):e259. doi: 10.1002/mco2.259. eCollection 2023 Jun.

Abstract

Gene therapy, a medical approach that involves the correction or replacement of defective and abnormal genes, plays an essential role in the treatment of complex and refractory diseases, such as hereditary diseases, cancer, and rheumatic immune diseases. Nucleic acids alone do not easily enter the target cells due to their easy degradation in vivo and the structure of the target cell membranes. The introduction of genes into biological cells is often dependent on gene delivery vectors, such as adenoviral vectors, which are commonly used in gene therapy. However, traditional viral vectors have strong immunogenicity while also presenting a potential infection risk. Recently, biomaterials have attracted attention for use as efficient gene delivery vehicles, because they can avoid the drawbacks associated with viral vectors. Biomaterials can improve the biological stability of nucleic acids and the efficiency of intracellular gene delivery. This review is focused on biomaterial-based delivery systems in gene therapy and disease treatment. Herein, we review the recent developments and modalities of gene therapy. Additionally, we discuss nucleic acid delivery strategies, with a focus on biomaterial-based gene delivery systems. Furthermore, the current applications of biomaterial-based gene therapy are summarized.

摘要

基因治疗是一种涉及纠正或替换有缺陷和异常基因的医学方法,在治疗复杂和难治性疾病,如遗传性疾病、癌症和风湿免疫性疾病方面发挥着重要作用。由于核酸在体内容易降解以及靶细胞膜的结构,单纯的核酸不容易进入靶细胞。将基因导入生物细胞通常依赖于基因递送载体,如腺病毒载体,其常用于基因治疗。然而,传统病毒载体具有很强的免疫原性,同时也存在潜在的感染风险。近年来,生物材料作为高效的基因递送载体受到关注,因为它们可以避免与病毒载体相关的缺点。生物材料可以提高核酸的生物稳定性和细胞内基因递送的效率。这篇综述聚焦于基因治疗和疾病治疗中基于生物材料的递送系统。在此,我们综述了基因治疗的最新进展和方式。此外,我们讨论了核酸递送策略,重点是基于生物材料的基因递送系统。此外,还总结了基于生物材料的基因治疗的当前应用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/78c3/10239531/f73be5cd8744/MCO2-4-e259-g001.jpg

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验