Gene therapy of the rheumatic diseases: 1998 to 2008.
Gene therapy of the rheumatic diseases: 1998 to 2008.
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DOI：
10.1186/ar2563
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发表时间：
2009
影响因子：
4.9
通讯作者：
Robbins PD
中科院分区：
文献类型：
--
作者：
Evans CH;Ghivizzani SC;Robbins PD
在关节炎研究启动的十年中，基因治疗在风湿性疾病中的应用经历了与整个基因治疗领域相同的变迁。概念和技术都有进步，临床试验的数量也有所增加。然而，资金一直是不可靠的，在人体试验中，包括一个关节炎基因治疗试验中的少数高调死亡，为怀疑者提供了弹药。尽管如此，在一些应用中取得了稳步进展，包括类风湿性关节炎和骨关节炎，干燥综合征和狼疮。类风湿性关节炎的临床试验已经进展到第二阶段，并提供了可能的疗效的第一个一瞥。目前正在进行两项关于骨关节炎的第一阶段方案。该原理已在干燥综合征和狼疮的动物模型中得到证实。对于某些适应症，发展基因疗法的主要技术障碍似乎已基本克服。将这些进展转化为有效的遗传药物所需的转化研究需要持续的资金和持续的努力。
During the decade since the launch of Arthritis Research, the application of gene therapy to the rheumatic diseases has experienced the same vicissitudes as the field of gene therapy as a whole. There have been conceptual and technological advances and an increase in the number of clinical trials. However, funding has been unreliable and a small number of high-profile deaths in human trials, including one in an arthritis gene therapy trial, have provided ammunition to skeptics. Nevertheless, steady progress has been made in a number of applications, including rheumatoid arthritis and osteoarthritis, Sjögren syndrome, and lupus. Clinical trials in rheumatoid arthritis have progressed to phase II and have provided the first glimpses of possible efficacy. Two phase I protocols for osteoarthritis are under way. Proof of principle has been demonstrated in animal models of Sjögren syndrome and lupus. For certain indications, the major technological barriers to the development of genetic therapies seem to have been largely overcome. The translational research necessary to turn these advances into effective genetic medicines requires sustained funding and continuity of effort.
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DOI：
10.1099/vir.0.82603-0
发表时间：
2007-06
期刊：
The Journal of general virology
影响因子：
--
作者：
Adriaansen J;Fallaux FJ;de Cortie CJ;Vervoordeldonk MJ;Tak PP
通讯作者：
Tak PP
影响因子：
46.9
作者：
Dalum, I;Butler, DM;Mouritsen, S
通讯作者：
Mouritsen, S
影响因子：
5.1
作者：
Fellowes, R;Etheridge, CJ;Woo, P
通讯作者：
Woo, P
影响因子：
6
作者：
Fernandes, J;Tardif, G;Pelletier, JP
通讯作者：
Pelletier, JP
影响因子：
4.2
作者：
Apparailly, F;Millet, V;Jorgensen, C
通讯作者：
Jorgensen, C