摘要
肌肉骨骼疾病是一个主要的公共卫生问题。在美国,约有6600万人因肌肉骨骼损伤而就医,目前的医疗费用估计为每年8,730亿美元。尽管在药物,植入材料和外科技术方面取得了进展,但仍然存在对成功治疗具有挑战性的肌肉骨骼损伤和病理状况的未满足的临床需求,特别是在受损的生物环境中。通过基因疗法的组织工程试图提供替代治疗策略以解决与常规方法相关的缺陷。编码具有治疗或再生特性的蛋白质的特定靶基因向疾病环境中的靶细胞和组织的转移允许它们在感兴趣的位点特异性地持续产生和释放。基于基因疗法的治疗在各种疾病的临床管理中成功的报道越来越多,这提供了真正的乐观主义,即类似的方法可以适用于肌肉骨骼疾病的主流临床应用。在临床前研究中,基因疗法已成功用于治疗软骨,骨骼,骨骼肌,肌腱,韧带和椎间盘损伤。此外,基因治疗正在临床试验中评估其在骨关节炎中的安全性和治疗潜力。本综述将专门针对基因治疗的临床潜力,临床前数据和未来障碍,以成为治疗骨折不愈合和难以修复骨关节软骨,关节软骨修复和骨关节炎的可行临床实体
关键词: 基因治疗,病毒载体,组织工程,骨愈合,软骨修复,骨关节炎。
Current Gene Therapy
Title:Gene Therapy to Enhance Bone and Cartilage Repair in Orthopaedic Surgery
Volume: 18 Issue: 3
关键词: 基因治疗,病毒载体,组织工程,骨愈合,软骨修复,骨关节炎。
摘要: Musculoskeletal conditions are a major public health problem. Approximately 66 million individuals seek medical attention for a musculoskeletal injury in the United States, with current medical costs being estimated at $873 billion annually. Despite advances in pharmaceuticals, implant materials and surgical techniques, there remains an unmet clinical need for successful treatment of challenging musculoskeletal injuries and pathologic conditions, particularly in the setting of compromised biological environments. Tissue engineering via gene therapy attempts to provide an alternative treatment strategy to address the deficits associated with conventional approaches. The transfer of specific target genes coding for proteins with therapeutic or regenerative properties to target cells and tissues in the disease environment allows for their sustained production and release specifically at the site of interest. The increasing reports of success with gene therapy-based treatments in the clinical management of a variety of diseases provide genuine optimism that similar methods can be adapted for mainstream clinical application in musculoskeletal disorders. In preclinical studies, gene therapy has been successfully used to treat cartilaginous, bone, skeletal muscle, tendon, ligament and intervertebral disk injuries. In addition, gene therapy is being assessed in clinical trials for its safety and therapeutic potential in osteoarthritis. This review will specifically address the clinical potential, preclinical data and future hurdles for gene therapy to be a viable clinical entity for the treatment of fracture nonunion and difficult bone repair scenarios, articular cartilage repair and osteoarthritis.
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Cite this article as:
Gene Therapy to Enhance Bone and Cartilage Repair in Orthopaedic Surgery, Current Gene Therapy 2018; 18 (3) . https://dx.doi.org/10.2174/1566523218666180410152842
DOI https://dx.doi.org/10.2174/1566523218666180410152842 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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