骨关节炎的新疗法:基因疗法。

IF 5.1 4区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Precision Clinical Medicine Pub Date : 2023-06-01 DOI:10.1093/pcmedi/pbad014
Xinyu Li, Leyao Shen, Zhenghan Deng, Zeyu Huang
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引用次数: 0

摘要

骨关节炎是一种复杂的退行性疾病,影响整个关节组织。目前,骨关节炎的非手术治疗主要是缓解疼痛。虽然终末期骨关节炎可以通过关节置换术治疗,但与手术相关的健康和经济成本迫使人们寻找其他非手术治疗方法来延缓骨关节炎的进展并促进软骨修复。与传统治疗方法不同,基因治疗方法允许治疗蛋白在特定部位持久表达。在这篇综述中,我们总结了骨关节炎基因治疗的历史,概述了常见的表达载体(非病毒,病毒),基因传递(转录因子,生长因子,炎症相关细胞因子,非编码rna)和基因传递模式(直接传递,间接传递)。重点介绍CRISPR/Cas9基因编辑技术在骨关节炎中的应用及发展前景。最后,我们确定目前的问题和可能的解决方案,在骨关节炎基因治疗的临床翻译。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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New treatment for osteoarthritis: Gene therapy.

Osteoarthritis is a complex degenerative disease that affects the entire joint tissue. Currently, non-surgical treatments for osteoarthritis focus on relieving pain. While end-stage osteoarthritis can be treated with arthroplasty, the health and financial costs associated with surgery have forced the search for alternative non-surgical treatments to delay the progression of osteoarthritis and promote cartilage repair. Unlike traditional treatment, the gene therapy approach allows for long-lasting expression of therapeutic proteins at specific sites. In this review, we summarize the history of gene therapy in osteoarthritis, outlining the common expression vectors (non-viral, viral), the genes delivered (transcription factors, growth factors, inflammation-associated cytokines, non-coding RNAs) and the mode of gene delivery (direct delivery, indirect delivery). We highlight the application and development prospects of the gene editing technology CRISPR/Cas9 in osteoarthritis. Finally, we identify the current problems and possible solutions in the clinical translation of gene therapy for osteoarthritis.

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来源期刊
Precision Clinical Medicine
Precision Clinical Medicine MEDICINE, RESEARCH & EXPERIMENTAL-
CiteScore
10.80
自引率
0.00%
发文量
26
审稿时长
5 weeks
期刊介绍: Precision Clinical Medicine (PCM) is an international, peer-reviewed, open access journal that provides timely publication of original research articles, case reports, reviews, editorials, and perspectives across the spectrum of precision medicine. The journal's mission is to deliver new theories, methods, and evidence that enhance disease diagnosis, treatment, prevention, and prognosis, thereby establishing a vital communication platform for clinicians and researchers that has the potential to transform medical practice. PCM encompasses all facets of precision medicine, which involves personalized approaches to diagnosis, treatment, and prevention, tailored to individual patients or patient subgroups based on their unique genetic, phenotypic, or psychosocial profiles. The clinical conditions addressed by the journal include a wide range of areas such as cancer, infectious diseases, inherited diseases, complex diseases, and rare diseases.
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