骨关节炎的基因治疗:进展与展望。

IF 2.8 4区 医学 Q2 PHARMACOLOGY & PHARMACY
Anjali Rana, Rishabha Malviya, Shivam Rajput, Sathvik Belagodu Sridhar, Javedh Shareef
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引用次数: 0

摘要

骨关节炎影响身体的一些关节,包括手的远端指间关节、膝盖和臀部。这种被称为退行性骨关节炎的复杂疾病会影响身体的每个关节。由于骨关节炎对负重关节的影响有限,并且没有实质性的关节外症状,因此骨关节炎非常适合在受影响的关节内进行靶向基因治疗。本文讨论了该领域的历史,所使用的基因,基因传递技术(直接与间接),以及最流行的表达载体(病毒与非病毒)。我们使用知名的数据库进行了全面的文献综述,包括Scopus、ScienceDirect、PubMed和谷歌Scholar。这项研究的重点是过去十年发表的研究,以确保纳入最近的进展。本文探讨了一系列用于治疗骨关节炎的基因治疗策略。重点是确定近年来出现的创新和临床相关的方法。通过回顾最新的数据,这项工作旨在提供一个详细的概述,基因治疗作为一种潜在的治疗途径来管理和可能逆转骨关节炎的症状和进展。骨关节炎(OA)基因治疗的目标是通过将基于基因的治疗剂以一种受调节的、特定部位的、长期的方式引入患处来修复受损的软骨。滑膜和软骨是两个可能进行基因转移的关节内部位。骨关节炎最先进的基因治疗方法是滑膜局部基因转移技术。IL-1是骨性关节炎软骨损失的关键调节剂,IL-1受体拮抗剂(IL-1Ra)基因转移在三种动物模型中有效治疗骨性关节炎。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Gene Therapy for the Treatment of Osteoarthritis: Advances and Prospective.

Osteoarthritis affects some joints in the body, including the hand's distal interphalangeal joints, knees, and hips. The complex disease known as degenerative osteoarthritis affects every joint in the body. Due to its limited influence on weight-bearing joints and absence of substantial extra-articular symptoms, osteoarthritis is well-suited for targeted gene therapy within the affected joints. The article discusses the field's history, the genes utilised, the techniques of gene delivery (direct vs. indirect), and the most prevalent expression vectors (viral vs. non-viral). A comprehensive literature review was conducted using reputable databases, including Scopus, ScienceDirect, PubMed, and Google Scholar. The study focused on research published over the past decade to ensure the inclusion of recent advancements. This article explores a range of gene therapy strategies developed for the treatment of osteoarthritis. Emphasis was placed on identifying innovative and clinically relevant approaches that have emerged in recent years. By reviewing the most current data, this work aims to provide a detailed overview of the evolving landscape of gene therapy as a potential therapeutic avenue for managing and possibly reversing osteoarthritis symptoms and progression. The goal of gene therapy for osteoarthritis (OA) is to repair damaged cartilage by introducing gene-based therapeutic agents to the affected area in a regulated, site-specific, long-term manner. The synovium and cartilage are two possible intra- articular sites for gene transfer. The most advanced gene therapy method for osteoarthritis is the local gene transfer to synovium technique. IL-1 is a key modulator of cartilage loss in OA, and IL-1 receptor antagonist (IL-1Ra) gene transfer is effective in treating OA in three animal models.

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来源期刊
CiteScore
6.30
自引率
0.00%
发文量
302
审稿时长
2 months
期刊介绍: Current Pharmaceutical Design publishes timely in-depth reviews and research articles from leading pharmaceutical researchers in the field, covering all aspects of current research in rational drug design. Each issue is devoted to a single major therapeutic area guest edited by an acknowledged authority in the field. Each thematic issue of Current Pharmaceutical Design covers all subject areas of major importance to modern drug design including: medicinal chemistry, pharmacology, drug targets and disease mechanism.
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