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类风湿关节炎的基因治疗

Gene therapy for rheumatoid arthritis.

作者信息

Gouze J N, Ghivizzani S C, Gouze E, Palmer G D, Betz O B, Robbins P D, Evans C H, Herndon J H

机构信息

Department of Orthopedic Surgery, Harvard Medical School, Boston, MA, USA.

出版信息

Hand Surg. 2001 Dec;6(2):211-9. doi: 10.1142/s0218810401000709.

Abstract

Advances in understanding the biology of rheumatoid arthritis (RA) have opened new therapeutic avenues. One of these, gene therapy, involves the delivery to patients of genes encoding anti-arthritic proteins. This approach has shown efficacy in animal models of RA, and the first human, phase I trial has just been successfully completed. Hand surgery featured prominently in this pioneering study, as a potentially anti-arthritic gene encoding the interleukin-1 receptor antagonist was transferred to the metacarpophalangeal joints of subjects with RA one week before total joint arthroplasty. This study has confirmed that it is possible to transfer genes safely to human joints. It should pave the way for additional application of gene therapy to arthritis and other orthopaedic conditions.

摘要

对类风湿性关节炎(RA)生物学特性认识的进展开辟了新的治疗途径。其中之一的基因疗法,涉及将编码抗关节炎蛋白的基因传递给患者。这种方法在RA动物模型中已显示出疗效,并且首次人体I期试验刚刚成功完成。在这项开创性研究中,手部手术占据重要地位,因为在全关节置换术前一周,将编码白细胞介素-1受体拮抗剂的一种潜在抗关节炎基因转移到了RA患者的掌指关节。这项研究证实了将基因安全转移到人体关节是可行的。它应为基因疗法在关节炎和其他骨科疾病中的进一步应用铺平道路。

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