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[前沿] Moving Gene Therapy Forward With Mobile DNA

[前沿] Moving Gene Therapy Forward With Mobile DNA

前沿

原创与否 -
Moving Gene Therapy Forward With Mobile DNA
ScienceDaily (May 9, 2009) — Gene therapy is the introduction of genetic material into a patient's cells resulting in a cure or a therapeutic effect. In recent years, it has been shown that gene therapy is a promising technology to treat or even cure several fatal diseases for which there is no attractive alternative therapy.

Gene therapy can be used for hereditary diseases, but also for other diseases that affect heart, brain and even for cancer. Indeed, recent results suggest that gene therapy can be beneficial for patients suffering from aggressive brain cancer that would otherwise be lethal.

A safe delivery of the genes?

Despite the overall progress, there is still a need to develop improved and safer approaches to deliver genes into cells. The success of gene therapy ultimately depends on these gene delivery vehicles or vectors. Most vectors have been derived from virusses that can be tailor-made to deliver therapeutic genes into the patients' cells. However, some of these viral vectors can induce side-effects, including cancer and inflammation.

Marinee Chuah, Thierry VandenDriessche, Eyayu Belay and their fellow VIB researchers at K.U. Leuven in collaboration with Zsuzsanna Iszvak and Zoltan Ivics and colleagues at the Max Delbrück Center in Berlin (Germany) have now developed a new non-viral approach that overcomes some of the limitations associated with viral vectors.

Lessons from evolution

Using the principles of evolution and natural selection, that were initially conceived by Charles Darwin, they have now developped an efficient and safe gene delivery approach based on non-viral genetic elements, called transposons. Transposons are mobile DNA elements that can integrate into 'foreign' DNA via a 'cut-and-paste' mechanism. In a way they are natural gene delivery vehicles. The researchers constructed the transposons in such a way that they can carry the therapeutic gene into the target cell DNA. Doing so, they obviate the need to rely on viral vectors.

'We show for the first time that it is now possible to efficiently deliver genes into stem cells, particularly those of the immune system, using non-viral gene delivery', says Marinee Chuah. 'Many groups have tried this for many years but without success. We are glad that we could now overcome this hurdle' claims Thierry VandenDriessche. Zsuzsanna Izsvak and Zoltan Ivics concur: 'This transposon technology may greatly simplify the way gene therapy is conducted, improve its overall safety and reduce the costs'.

The VIB researchers are further testing this technology to treat specific diseases including cancer and genetic disorders, in anticipation of moving forward and treat patients suffering from these diseases.

http://www.sciencedaily.com/releases/2009/05/090503132615.htm


基因治疗已经进展到可移动DNA。

ScienceDaily ( 2009年5月9日)报道:基因疗法是通过将遗传物质注入病人的细胞从而起到治疗作用的一种治疗方法。近年来的资料显示,基因治疗是一种很有前途的治疗技术。它甚至可以治愈一些目前无药可救的致命性疾病。

基因疗法除了可用于遗传性疾病,而且还可以用于其他影响心、脑等疾病,甚至可用于治疗癌症。事实上,最近的结果表明,基因疗法可以用于治疗致命性的浸润性脑癌。

一项更加安全的基因注入方式?

虽然基因治疗的整体取得了很大的进展,但人们仍然需要开发更好的和更安全的将基因注入细胞的注射方式。基因治疗能否成功最终取决于这些基因的运输载体。绝大部分载体都来自病毒,这些病毒可将治疗性基因导入患者的细胞。然而无论如何,其中一部分病毒载体可诱导产生相关的副作用,包括引发癌症和炎症。

在堪萨斯鲁汶的研究人员Marinee Chuah ,Thierry VandenDriessche,Eyayu Belay和他们的同事与在柏林的马克斯德尔布吕克中心的Zsuzsanna Iszvak,Zoltan Ivics和他们同事已经联手开发出一种新的无病毒的基因导入方法。这种方法可以克服病毒载体无法克服的限制。

进化的情况

根据查尔斯达尔文最初提出设想的进化论和自然选择,现在他们开发了一种安全有效非病毒的基因注入方式,就是所谓的转位子。转位子是一种移动的DNA分子,它通过可以通过“剪切和粘贴”机制融入异体的DNA分子。从某种程度上说,它们是天然的基因运载工具。研究人员构建了转位子,通过转位子携带治疗基因进入靶细胞的DNA 。这样注入细胞,他们就可以不必依赖于病毒载体。

Marinee Chuah 说:“我们首次展示了使用的非病毒基因传递方式使基因有效的植入干细胞,特别是进入那些免疫系统”。Thierry VandenDriessche说 :“许多团队研究了许多年,但都没有成功。我们高兴地宣布我们现在可以克服这一障碍”。 Zsuzsanna Izsvak和Zoltan Ivics都认为:“这项导入技术大大简化了基因治疗的方式,同时提高其总体安全性并降低了成本。”

VIB的研究人员正在进一步研究这一技术来治疗包括癌症和遗传性疾病等一系列疾病,预计患有这些疾病的患者将会因此获益。


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