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English

In 2009 researchers at San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy performed a similar pilot study, and it and the virus stopped the disease in its tracks. Then last month, the institute  reported success in using lentivirus to treat metachromatic leukodystrophy, another enzyme deficiency disorder.But despite a number of promising trials, the Food and Drug Administration still has not approved a gene therapy drug for market in the U.S."What has been missing, oddly, is the ability of industry to come forth with large reproducible clinical trials," Mulligan says.

Chinese

2009年,意大利米兰的San Raffaele-Telethon基因疗法研究院的研究人员们进行了类似的导航研究,病毒使症状消失了。 之后在上个月,该研究院报告在使用慢病毒治疗异染性脑白质营养不良时也取得了成功,这是另一种酶缺乏症。 但尽管已有不少试验取得了良好的效果,食品药品监督管理局仍然没有批准任何一种基因疗法的药物进入美国市场 。 "