打开APP

研究人员发现GSK一活性成分在动物模型上能延缓重症肌无力病情

  1. GSK
  2. TDP-43
  3. 重症肌无力

来源:生物谷 2013-12-23 09:24

2013年12月20日讯 /生物谷BIOON/ --最近来自宾州大学的一个研究团队发现能够在动物模型上减缓神经元功能异常来抑制ALS病情蔓延。这一发现可能为彻底治愈这一疾病提供新的思路。此前该团队研究发现一个名为TDP-43的基因与ALS有关。在果蝇模型上研究人员发现TDP-43与一种压力颗粒相关,据此研究人员猜测ALS患者体内TDP-43的表达量可能出现异常。因此研究人员利用来自葛兰素史克的一种原本用来降低eIF2α磷酸化的成分来处理小鼠模型,结果发现这种成分降低了小鼠神经元的死亡率。这一成果发表在Nature Genetics杂志上。

路格里克氏病又称肌萎缩性脊髓侧索硬化症,是一种严重的神经退行性疾病,它能够导致脑部运动神经元凋亡并最终导致机体绝大多数运动机能丧失。这种疾病目前尚无法治愈,目前FDA仅批准一种名为riluzole的药物治疗该疾病,但这种药物也仅仅只能延长患者近三个月的生命。(生物谷Bioon.com)

详细英文报道:

A team headed by researchers at the University of Pennsylvania has figured out a way to reduce the toxicity of Lou Gehrig's disease by slowing neuron dysfunction in animal models. The discovery could offer a new way to treat the disease, also known as amyotrophic lateral sclerosis, or ALS.

Previous studies by the Penn team found that TDP-43, a gene linked with ALS, interacts with a gene called ataxin-2, which on its own is a gene whose mutations cause human degenerative disease.

Using fruit fly models of the disease, investigators found that genes controlling cellular structures known as stress granules, which act as holding pens for RNA and proteins when cells are under stress, also modify TDP-43 toxicity. Previous research has suggested that ALS patients may have abnormal buildups of these stress granule components. The team found that when a set of genes that promote stress granules were expressed, it correlated with an increased in the toxic activity of TDP-43.

To test reversing TDP-43 toxicity, the researchers used a compound developed by GlaxoSmithKline ($GSK) that reduces eIF2α phosphorylation, which is associated with the formation of stress granules. Flies expressing TDP-43 that were fed the compound showed greater physical strength and more climbing ability than those that were not treated with the compound. When tested in rat neuron cells expressing TDP-43, researchers found that the compound reduced the risk of cell death.

Researchers say the findings, which are published in Nature Genetics, could provide the groundwork for new therapies to treat ALS.

ALS is a fatal neurodegenerative disease that gradually degrades motor neurons in the brain, causing the loss of major motor functions, including walking, speaking, swallowing and breathing. The disease is usually fatal within 5 years of diagnosis. There is no cure for ALS and, so far, only one FDA-approved drug, riluzole, which extends the lifespans of patients by an average of only three months.

 

版权声明 本网站所有注明“来源:生物谷”或“来源:bioon”的文字、图片和音视频资料,版权均属于生物谷网站所有。非经授权,任何媒体、网站或个人不得转载,否则将追究法律责任。取得书面授权转载时,须注明“来源:生物谷”。其它来源的文章系转载文章,本网所有转载文章系出于传递更多信息之目的,转载内容不代表本站立场。不希望被转载的媒体或个人可与我们联系,我们将立即进行删除处理。

87%用户都在用生物谷APP 随时阅读、评论、分享交流 请扫描二维码下载->