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基因治疗遗传性眼病进入新阶段

Gene therapy sets stage for new treatments for inherited blindness

Veterinary vision scientists at the University of Pennsylvania have safely and successfully used a viral vector in targeting a class of photoreceptors of the retina called rods, a critical first step in developing gene therapies for inherited blindness caused by rod degeneration.

In this study, the viral vector, or missile that carries the genetic material designed to correct a DNA mutation, was not intended to treat a disease but to demonstrate through the use of a fluorescent protein that a safe and effective viral cocktail could be delivered inside rod cells.

The next major challenge that vision researchers face is to target these photoreceptor cells for treatment, as the majority of retinal degenerative diseases are caused by mutations that damage these cells. Photoreceptors are the cells in the retina responsible for capturing light and transforming it into an electrical signal that will be interpreted by the brain.

A consortium of researchers from Penn and the University of Florida used a specific adeno-associated virus, AAV2/5, to carry the gene of a green fluorescent protein. The scientists tested whether three different promoters, pieces of DNA that play the role of a switch, could turn on the production of the green fluorescent protein in rods in dogs. Two of the three viral cocktails were able to turn on the production of the green fluorescent protein specifically in rods, while the third viral cocktail targeted rods, cones and other retinal cells; however only the proper concentration of each viral vector proved to be just right. Efficient and specific rod transduction, together with preservation of retinal structure, was achieved with both mOP and hGRK1 promoters when viral titers in the order of 1011 vg/ml were used.

'Now that we've demonstrated this type of delivery can be accomplished, ' said William A. Beltran, assistant professor of ophthalmology at Penn's School of Veterinary Medicine and director of the study, 'the next step is to target common rod degenerations using canine models that mimic the human diseases. The delivery of a viral vector, coupled with a rod-specific promoter is likely to be the safest and most efficient approach to correct diseases such as X-linked and autosomal dominant retinitis pigmentosa, both of which lead to complete blindness.'

A form of childhood blindness called RPE65-Leber's congenital amaurosis, LCA, is the first - and to this date the only - inherited retinal disease for which corrective gene therapy is currently being tested in three on-going clinical trials. This treatment was first developed and perfected in dogs by some of the scientists who participated in the present study before its use in human patients.

The study appears in the current issue of the Journal of Gene Therapy.



Source: Penn: Office of University Communications
这篇文章太专业了,用翻译软件很难看懂。

好像是说视网膜杆状细胞退化疾病(rod degeneration)
飞狐兄.看不懂啊.要翻译过来才能看懂啊.
是针对RP的好消息吗?
以下用翻译器翻译,看不太懂——
基因治疗遗传性失明设置为新的治疗阶段

在宾夕法尼亚大学兽医视觉科学家已安全和成功地用于瞄准一个名为棒的视网膜,一个关键的发展为棒豆状核变性引起的遗传性致盲基因疗法的第一步感光类的病毒载体。

在这项研究中,病毒载体,或导弹,携带的遗传物质,以正确的DN*突变,不是为了治疗疾病,而是说明通过一种荧光蛋白的一种安全有效的病毒鸡尾酒内可交付使用视杆细胞。

下一个主要挑战视觉研究人员面临的是针对这些感光细胞治疗视网膜变性的疾病多数是由于基因突变,损害这些细胞。在视网膜光感受器捕捉光线转化成电子信号,将由它负责解释大脑细胞。

一个来自宾州大学的研究财团和佛罗里达大学使用一个特定的腺相关病毒,AAV2 / 5,携带的绿色荧光蛋白基因。科学家测试是否三种不同的推动者,对DNA*段中扮演一个开关的作用,可以把对狗棒在绿色荧光蛋白的生产。三个病毒鸡尾酒两人能够打开具体棒的绿色荧光蛋白的生产,而第三杆病毒鸡尾酒针对性,圆筒和其他视网膜细胞,不过只是每个病毒载体证明是正确的浓度恰到好处。高效和具体棒转导,与视网膜结构保存起来,既实现了拖把和hGRK1发起人时的1011 vg的秩序病毒滴度/ ml的使用。

'现在我们已经证明了这一点交货类型可以完成,'威廉A贝尔特兰说,眼科助理教授宾夕法尼亚大学兽医学和研究主任学校,'下一步目标是共同使用犬杆变性模型模拟人类疾病。一个病毒载体传递,与一杆特异性启动子耦合可能是最安全和最有效的方法来纠正,如X -连锁和常染色体显性遗传视网膜色素变性疾病,它们都导致完全失明。

一个儿童失明的形式要求的RPE65 -雷伯氏先天性黑朦,LCA的,是第一个 - 并且这个日期是唯一的 - 遗传性视网膜疾病的基因治疗是纠正目前正在测试中的三个正在进行临床试验。这种治疗是首先开发并完善由犬的科学家谁在人前,参与研究的病人使用目前一些。

这项研究发表在基因治疗学当前的问题。



来源:佩恩:大学传讯公关处
是啊,看不懂呀,不知道风之子版主什么时候有空翻译一下?
谢谢楼主提供分享!呵呵,大概没看懂。
我想,RP这个病魔快完完了,嘎嘎!!
我们不是病人,而是战士!!!
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