Advancements in adeno-associated viral gene therapy approaches: exploring a new horizon.
Advancements in adeno-associated viral gene therapy approaches: exploring a new horizon.
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与腺相关的病毒基因疗法的进步：探索新的视野。
DOI：
10.3410/m3-17
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发表时间：
2011
期刊：
影响因子：
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通讯作者：
Vervoordeldonk MJ
中科院分区：
文献类型：
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作者：
Aalbers CJ;Tak PP;Vervoordeldonk MJ
基因治疗是一种很有前途的新的治疗策略，已经在各种疾病中进行了探索，从癌症到血友病，从眼部疾病到自身免疫性疾病等。基因转移方法的概念证明已经在100多项疾病动物模型的研究中显示，尽管只有少数正在开发用于临床应用。到目前为止，美国食品和药物管理局和欧洲药品管理局还没有批准任何病毒性人类基因治疗产品用于销售，但在美国和欧洲发生的与基因相关的研究和开发的数量继续以快速的速度增长。本文综述了以腺相关病毒为载体的病毒基因治疗领域的发展现状，特别是关节炎。对于类风湿性关节炎，以及在较小程度上对于其他免疫相关的炎症性疾病，已经在临床前水平研究了几种细胞和基因转移方法，并且一些已经在临床试验中实施。最后，通过其临床应用的病毒基因治疗的发展过程中所面临的潜力和障碍进行了讨论。
Gene therapy is a promising new therapeutic strategy that has been explored in a wide variety of diseases, ranging from cancer to hemophilia, and ocular disorders to autoimmune diseases, among others. Proof of concept of gene transfer approaches has been shown in over 100 studies of animal models of disease, although only a few are under development for clinical application. The US Food and Drug Administration and the European Medicines Agency have not approved any viral human gene therapy products for sale so far, but the amount of gene-related research and development occurring in the United States and Europe continues to grow at a fast rate. This review summarizes the current status of developments in the field of viral gene therapy using adeno-associated virus as a vector, with a special focus on arthritis. For rheumatoid arthritis, and to a lesser extent for other immune-related inflammatory disorders, several cell and gene transfer approaches have been investigated at the preclinical level and a few have been implemented in clinical trials. Finally, both the potential and the hurdles that are faced during development of a viral gene therapy through to its clinical application are discussed.