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New CRISPR tool alters RNA for wider gene editing applications

RNA-based diseases like Fragile X, myotonic dystrophy and ALS could be treated with this technique.
Rob LeFebvre, @roblef
October 25, 2017
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The CRISPR gene editing technique can be used for all sorts of amazing things by targeting your DNA. Scientists are using it in experimental therapies for ALS and Huntington's disease, ways to let those with celiac disease process gluten proteins and possibly assist in more successful birth rates. Now, according to a paper published in Science, researchers have found a way to target and edit RNA, a different genetic molecule that has implications in many degenerative disorders like ALS.

Apparently, edits with this new tool (CRISPR-Cas13) can be safer as they don't result in permanent changes to your genetic makeup like other DNA-based CRISPR techniques can. This system, called REPAIR, works more efficiently in human cells, as well. RNA is implicated in various diseases like amyotrophic lateral sclerosis (ALS), myotonic dystrophy and Fragile X-associated tremor/ataxia syndrome (FXTAS), so fixing it could show positive results in treating these types of disorders.

"REPAIR presents a promising RNA editing platform with broad applicability for research, therapeutics, and biotechnology," wrote the researchers.

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