【RNA基因编辑技术 能治疗渐冻症和亨廷顿舞蹈病】

【RNA基因编辑技术 能治疗渐冻症和亨廷顿舞蹈病】常见的基因编辑技术CRISPR-Cas9只能修饰DNA,但无法处理基于RNA的疾病。近日,加州大学圣地亚哥分校的研究人员开发了一种面向RNA的新技术(RCas9),可以追踪体内特异的RNA序列,将RNA引导到靶RNA分子的匹配序列,固定患者肌肉细胞中破损的RNA靶标,使细胞更加健康。有望治疗遗传性ALS和亨廷顿病。

Gene editing technique could treat ALS and Huntington's disease

It might delay the onset of conditions that are normally inescapable.
Jon Fingas, @jonfingas

35m ago in Medicine
blue double helix models on background

blue double helix models on background

The most common gene editing technique, CRISPR-Cas9, only modifies DNA. That's helpful in most cases, but it means that you can't use it to tackle RNA-based diseases. Thankfully, that might not be a problem for much longer. After plenty of talk about editing RNA, researchers havedeveloped a new RNA-oriented technique (RCas9) that can correct the molecular errors which lead to diseases like hereditary ALS andHuntington's. The team achieved its feat using guide RNA that steers the editing enzyme toward a matching sequence of target RNA molecules. In the lab, it's highly effective. It fixed nearly all of the broken RNA targets in a patient's muscle cell sample, leaving cells that were far healthier.

The team achieved its feat using guide RNA that steers the editing enzyme toward a matching sequence of target RNA molecules. In the lab, it's highly effective -- it fixed nearly all of the broken RNA targets in muscle cells, leaving cells that were far healthier.

Of course, this is in lab conditions. The scientists readily admit that there's much more work to be done before gene editing could help in the field. More than anything, it would be difficult to send RCas9 to patient cells. The benign viruses you'd normally use for gene therapy just can't hold a full-fledged Cas9 payload; the researchers had to prune theirs to make it fit. And of course, there's the question of conducting trials and proving that the technique is safe.

Nonetheless, this could prove to be hugely important for treating these RNA diseases. Right now, there aren't any therapies that would stall the onset of diseases like ALS. This wouldn't likely represent a cure, but it could give patients full functionality for longer before the effects of their diseases set in.

https://www.engadget.com/2017/08/13/gene-editing-technique-could-treat-als-and-huntingtons-disease/


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