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骨关节炎的非病毒基因治疗

Non-viral Gene Therapy for Osteoarthritis.

作者信息

Uzieliene Ilona, Kalvaityte Ursule, Bernotiene Eiva, Mobasheri Ali

机构信息

Department of Regenerative Medicine, State Research Institute Centre for Innovative Medicine, Vilnius, Lithuania.

Research Unit of Medical Imaging, Physics and Technology, Faculty of Medicine, University of Oulu, Oulu, Finland.

出版信息

Front Bioeng Biotechnol. 2021 Jan 13;8:618399. doi: 10.3389/fbioe.2020.618399. eCollection 2020.

Abstract

Strategies for delivering nucleic acids into damaged and diseased tissues have been divided into two major areas: viral and non-viral gene therapy. In this mini-review article we discuss the application of gene therapy for the treatment of osteoarthritis (OA), one of the most common forms of arthritis. We focus primarily on non-viral gene therapy and cell therapy. We briefly discuss the advantages and disadvantages of viral and non-viral gene therapy and review the nucleic acid transfer systems that have been used for gene delivery into articular chondrocytes in cartilage from the synovial joint. Although viral gene delivery has been more popular due to its reported efficiency, significant effort has gone into enhancing the transfection efficiency of non-viral delivery, making non-viral approaches promising tools for further application in basic, translational and clinical studies on OA. Non-viral gene delivery technologies have the potential to transform the future development of disease-modifying therapeutics for OA and related osteoarticular disorders. However, further research is needed to optimize transfection efficiency, longevity and duration of gene expression.

摘要

将核酸递送至受损和患病组织的策略主要分为两个领域

病毒基因治疗和非病毒基因治疗。在这篇小型综述文章中,我们讨论了基因治疗在骨关节炎(OA)治疗中的应用,OA是最常见的关节炎形式之一。我们主要关注非病毒基因治疗和细胞治疗。我们简要讨论了病毒基因治疗和非病毒基因治疗的优缺点,并回顾了已用于将基因递送至滑膜关节软骨中关节软骨细胞的核酸传递系统。尽管由于其报道的效率,病毒基因递送更受欢迎,但人们已付出巨大努力来提高非病毒递送的转染效率,这使得非病毒方法成为在OA的基础、转化和临床研究中进一步应用的有前景的工具。非病毒基因递送技术有可能改变OA及相关骨关节疾病的疾病修饰疗法的未来发展。然而,需要进一步研究以优化转染效率、基因表达的持久性和持续时间。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5299/7838585/099bc18eec01/fbioe-08-618399-g0001.jpg

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