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针对椎间盘退变及相关脊柱疾病的基因治疗方法。

Gene therapy approach for disc degeneration and associated spinal disorders.

作者信息

Nishida Kotaro, Suzuki Teppei, Kakutani Kenichiro, Yurube Takashi, Maeno Koichiro, Kurosaka Masahiro, Doita Minoru

机构信息

Department of Orthopaedic Surgery, Kobe University Graduate School of Medicine, 7-5-1 Kusunoki-cho, Chuo-ku, Kobe, 650-0017, Japan.

出版信息

Eur Spine J. 2008 Dec;17 Suppl 4(Suppl 4):459-66. doi: 10.1007/s00586-008-0751-5. Epub 2008 Nov 13.

Abstract

Disc degeneration is deeply associated with many spinal disorders and thus has a significant clinical impact on society. The currently available surgical treatment often necessitates removing a pathological disc and spinal fusion. However, it is also well known that these surgical treatments have many potential problems including invasion and cost. Therefore, biological approaches for regenerating these pathological discs have received much attention. Gene therapy is one of these biological approaches. Gene therapy involves the transfer of genes to cells so the recipient cells express these genes and thereby synthesize the RNA and protein they encode in a continuous fashion. One of the significant advantages of gene therapy is that we can expect a lasting duration of biological effect which is potentially beneficial for most disc degeneration associated disorders, as they are, by nature, chronic conditions. Originally, gene therapy was mediated by viral vectors, but recent technological progress has enabled us to opt for non-virus-mediated gene therapy for the disc. Furthermore, the development of the RNA interference technique has enabled us to down-regulate a specific gene expression in the disc opening the door for a new generation of intradiscal gene therapy.

摘要

椎间盘退变与许多脊柱疾病密切相关,因此对社会具有重大的临床影响。目前可用的手术治疗通常需要切除病变椎间盘并进行脊柱融合。然而,众所周知,这些手术治疗存在许多潜在问题,包括侵袭性和成本。因此,再生这些病变椎间盘的生物学方法受到了广泛关注。基因治疗就是其中一种生物学方法。基因治疗涉及将基因转移到细胞中,使受体细胞表达这些基因,从而持续合成它们编码的RNA和蛋白质。基因治疗的一个显著优点是我们可以期待生物学效应的持久持续时间,这对于大多数与椎间盘退变相关的疾病可能是有益的,因为这些疾病本质上是慢性疾病。最初,基因治疗是由病毒载体介导的,但最近的技术进步使我们能够选择非病毒介导的椎间盘基因治疗。此外,RNA干扰技术的发展使我们能够下调椎间盘中特定基因的表达,为新一代椎间盘内基因治疗打开了大门。

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