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用于治疗与年龄相关的肌肉骨骼疾病的寡核苷酸疗法:成功与挑战

Oligonucleotide Therapeutics for Age-Related Musculoskeletal Disorders: Successes and Challenges.

作者信息

Nicholson Thomas A, Sagmeister Michael, Wijesinghe Susanne N, Farah Hussein, Hardy Rowan S, Jones Simon W

机构信息

MRC Versus Arthritis Centre for Musculoskeletal Ageing Research, Institute of Inflammation and Ageing, University of Birmingham, Birmingham B15 2TT, UK.

Institute for Metabolism and Systems Research, University of Birmingham, Birmingham B15 2TT, UK.

出版信息

Pharmaceutics. 2023 Jan 10;15(1):237. doi: 10.3390/pharmaceutics15010237.

Abstract

Age-related disorders of the musculoskeletal system including sarcopenia, osteoporosis and arthritis represent some of the most common chronic conditions worldwide, for which there remains a great clinical need to develop safer and more efficacious pharmacological treatments. Collectively, these conditions involve multiple tissues, including skeletal muscle, bone, articular cartilage and the synovium within the joint lining. In this review, we discuss the potential for oligonucleotide therapies to combat the unmet clinical need in musculoskeletal disorders by evaluating the successes of oligonucleotides to modify candidate pathological gene targets and cellular processes in relevant tissues and cells of the musculoskeletal system. Further, we discuss the challenges that remain for the clinical development of oligonucleotides therapies for musculoskeletal disorders and evaluate some of the current approaches to overcome these.

摘要

与年龄相关的肌肉骨骼系统疾病,包括肌肉减少症、骨质疏松症和关节炎,是全球一些最常见的慢性病,临床上仍非常需要开发更安全、更有效的药物治疗方法。总体而言,这些疾病涉及多个组织,包括骨骼肌、骨骼、关节软骨和关节衬里内的滑膜。在本综述中,我们通过评估寡核苷酸修饰肌肉骨骼系统相关组织和细胞中候选病理基因靶点及细胞过程的成功案例,探讨寡核苷酸疗法应对肌肉骨骼疾病中未满足临床需求的潜力。此外,我们讨论了寡核苷酸疗法用于肌肉骨骼疾病临床开发中仍然存在的挑战,并评估了当前一些克服这些挑战的方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2794/9866666/4878c10c6333/pharmaceutics-15-00237-g001.jpg

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