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标题: 病人的皮肤细胞可以帮助研究人员将卵状黄斑变性治疗 [打印本页]

作者: 凤凰涅盘 时间: 2012-11-10 18:15 标题: 病人的皮肤细胞可以帮助研究人员将卵状黄斑变性治疗

本帖最后由凤凰涅盘于 2012-11-10 19:49 编辑

Nov. 8, 2012 – Contrary to its name, Best disease is not a condition anyone wants. Named after the German ophthalmologist Friedrich Best more than a century ago, it is an inherited form of macular degeneration that can cause significant and progressive loss of central vision in children and adults.

But thanks to a newly developed human model of the disease — derived from the skin of two affected patients — researchers have a new tool to study the condition and help develop treatment approaches that might save vision. The research advancement was funded by the Foundation Fighting Blindness and posted today in the journal Human Molecular Genetics.

Dr. David Gamm, director of the McPherson Eye Research Institute at the University of Wisconsin-Madison, used an innovative technique to reprogram the patients’ skin cells into what are known as induced pluripotent stem cells, or iPSC. He then coaxed the iPSC forward to become retinal pigment epithelial (RPE) cells.

RPE cells play a critical supporting role in the retina, providing nutrition and waste disposal for photoreceptors, the cells that make vision possible. RPE cells are directly affected by Best disease, and when they become dysfunctional, photoreceptors in the central region of the retina known as the macula are progressively lost.

By studying the affected RPE cells in a dish, Dr. Gamm and his team were able to observe and understand critical elements of the disease process, including their ability to pump fluid which leads to the build-up of cysts. They also saw how the RPE cells did not effectively dispose of the tips of photoreceptors, which are shed every night during sleep. Additionally, the researchers noted that certain proteins — including rhodopsin, a pigment in photoreceptors that is critical for vision — were not being properly degraded by the RPE cells.

Studying Best disease in a laboratory dish using RPE cells derived from iPSC has many advantages and complements animal models of the condition. “It is a faster and less expensive process than using animal models,” Dr. Gamm says. “And, most important, it provides a human model of disease to help us understand how and why vision is lost in people. However, animal models remain essential in understanding disease and identifying potential cures, namely because they are a complete biological system.”

Dr. Gamm, a recent recipient of the Foundation’s Visionary Award, adds that the families’ perspectives on the research have been exciting for him. “One family came to my lab recently, and we spent an afternoon discussing their cells and their implications for them and their affected kids,” he says. “They are very happy to be involved in the research and our work toward treatments and cures.”

His laboratory is now using the families’ cells to evaluate potential vision-saving therapies.
http://www.blindness.org/index.p ... ;catid=68

作者: BOBOm 时间: 2012-11-10 19:16

谢谢凤凰涅盘的分享！

作者: 开心之硕 时间: 2012-11-10 19:30

本帖最后由开心之硕于 2012-11-10 19:33 编辑

Nov. 8, 2012 – Contrary to its name, Best disease is not a condition anyone wants. Named after the German ophthalmologist Friedrich Best more than a century ago, it is an inherited form of macular de ...
凤凰涅盘发表于 2012-11-10 18:15

主要介绍的一种研究方法，就是直接在实验室培养皿里面观察和研究致病细胞，然后寻求治疗方法，相对于运用动物模型更快捷，基本原理还是干细胞疗法。
下面我自己翻译的，其中有些可能不准确。
翻译：
   11.8 2012  与它的名字相反，卵黄状黄斑变性（Best Disease，直译则为最好的疾病）并不是人人想成为的状态。它是以一个多世纪之前的德国眼科医师Friedrich Best的名字而命名的，此种黄斑变性是一种遗传性黄斑退化疾病，能够引起成年人和儿童严重进行性的中央视野缺失。
   但是，所幸由于一种最新的发展起来的人类疾病模型（来自于两个受到影响的病人），研究者们有了一种新的方法来研究这种疾病，以帮助实现拯救视力的疗法。这项研究进展由美国抗盲基金会资助，并且宣告于《人类分子基因学》期刊上。
David Gamm博士是Wisconsin-Madison大学McPherson眼科研究学院的主席，他用一种使用一种创新技术对病人的皮肤细胞重新“编程”，使其变成IPS或者IPSC细胞。然后诱导iPSC细胞成为视网膜色素上皮细胞（RPE细胞）。
RPE 细胞在维持视网膜运作中发挥着关键的作用，它提供营养，处理感光细胞的排泄物（感光细胞就是能够产生视觉的细胞）。RPE细胞会被黄斑变性直接影响，当RPE细胞丧失功能时，视网膜中央部分的感光细胞就会渐进性的死亡丢失。
通过在杯盘中研究受影响的RPE细胞，Gamm博士和他的小组已经观测和理解了这种疾病进程的这些关键原理，如RPE细胞能够传输建立生物体细胞的液体营养成分。它们还观测到RPE细胞为何没有正常有效处理感光细胞的排泄物，这些排泄物在每天晚上睡觉时都在散步脱落。另外，研究者们注意到包含视紫质的蛋白质没有被RPE细胞正确降解（视紫质是感光细胞中的色素，对视野起到关键作用）。
在实验室杯盘里面，运用来自于iPSC细胞的RPE研究卵状黄斑变性，这样的研究方法有许多优势以及弥补运用有此类疾病的动物模型的不足。Gamm说到，“相对于运用动物模型，这样更加快速，更加便宜，最重要的是这样提供了一个人类疾病的模型，让我们理解视野在患病者中是如何以及怎样丢失的。但是，在理解和识别潜在的治疗方式的时候，动物模型仍然是必要的，仅仅因为他们是一个完整的生物体。”
      Gamm博士是基金会眼视觉奖项的得主，他另外说到，患者家族对待这项研究的态使他感到很激励。他说，“一个家族的成员来到我的实验室，我们花了一整个下午谈论他们的细胞，以及对他们以及他们孩子的影响，他们很高兴能够参与到这项研究中来，我们的工作的目的就是治疗和治愈他们。”
   他的实验室正在运用这个家族的细胞来评估这种治疗方法对视力维持的潜在疗效。

作者: 开心之硕 时间: 2012-11-10 19:36

1# 凤凰涅盘

凤凰，把题目中的那个“最好的疾病”改一下吧，我差了一下字典，实际上指的卵状黄斑变性，是翻译”Best Disease“的问题

作者: 凤凰涅盘 时间: 2012-11-10 19:48

4# 开心之硕
谢谢指教，英语多年不用了都忘了，现在都靠翻译软件。

作者: 开心之硕 时间: 2012-11-10 20:01

6# 凤凰涅盘

凤凰大哥，指教就不敢了，你为我们rp人作这么多，您才是功臣

作者: 祝福RP 时间: 2012-11-11 18:40

谢谢楼主分享辛苦了

作者: 祝福RP 时间: 2012-11-11 18:41

谢谢楼主分享辛苦了

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