Ivković A, Pascher A, Hudetz D, Jelić M, Haspl M, Windhager R, Pećina M
Department of Orthopaedic Surgery, School of Medicine University of Zagreb, Croatia.
Acta Chir Orthop Traumatol Cech. 2006 Apr;73(2):115-22.
The purpose of this article is to review the remarkable progress in the field of musculoskeletal system gene therapy. Since the introduction of this concept 15 years much of the preclinical and clinical data have emerged. The original target, rheumatoid arthritis, has been subjected to clinical phase II efficacy protocol, and osteoarthritis gene therapy efficacy is being thoroughly investigated in various animal models. The most promising area of research in this field however, is the tissue repair, because it doesn't require prolonged period of gene expression, local delivery is reasonably simple and it avoids substantial risk associated with systemic delivery, and levels of gene expression don't need to be so finely regulated. Gene transfer is successfully being used to aid the repair and regeneration of bone, cartilage, ligament tendon, meniscus and intervertebral disc. Other potential applications of gene therapy in musculoskeletal system include osteoporosis, aseptic loosening, genetic diseases and tumors. Highly encouraging data gained from these studies have confirmed that gene therapy is a promising therapeutic solution to treat various musculoskeletal system disorders.
本文旨在综述肌肉骨骼系统基因治疗领域取得的显著进展。自这一概念提出15年来，已涌现出大量临床前和临床数据。最初的靶点类风湿关节炎已进入临床II期疗效试验方案，骨关节炎基因治疗的疗效正在各种动物模型中进行深入研究。然而，该领域最有前景的研究方向是组织修复，因为它不需要长时间的基因表达，局部给药相对简单，避免了与全身给药相关的重大风险，且基因表达水平无需精确调控。基因转移已成功用于促进骨、软骨、韧带、肌腱、半月板和椎间盘的修复与再生。基因治疗在肌肉骨骼系统的其他潜在应用包括骨质疏松症、无菌性松动、遗传性疾病和肿瘤。从这些研究中获得的极具鼓舞性的数据证实，基因治疗是治疗各种肌肉骨骼系统疾病的一种有前景的治疗方法。
