“We got a little bit of rescue, but it wasn’t great,” said Wang.
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Vincent Dion simply wanted to understand how unstable, repetitive regions of the genome contract and in some cases expand, inciting disease.
He started by cutting across both strands of a set of repeats, then tried just nicking one strand.
The roadblocks reduced, somewhat, the tell-tale signs of the disease at five weeks, including myotonia.
However, the treatment did not stop the synthesis of expanded repeat RNAs or repair splicing in all muscle cells.
They used a deactivated version of Cas9 (d Cas9) as the physical barrier, with the help of a guide RNA to direct the “dead” endonuclease to the repeat-riddled gene. In this disease, triplet CTG repeats form RNA foci that sequester the splicing factor muscleblind, altering the transcriptome for the worse.
In muscle cells from people with myotonic dystrophy type 1, the approach significantly reduced the number of cells containing RNA foci and restored RNA splicing.By adding roadblocks—like felled trees dotting the hill—in the form of deactivated Cas9 enzyme, researchers at the University of Florida in Gainesville aim to grind this process to a halt, reducing levels of expanded repeat RNAs.[Courtesy of Eric Wang, Reproduced with permission.]Repeat tracts are dangerous, but they’re also a vulnerable spot, said Eric Wang of the University of Florida.“We ended up with this very surprising result,” said Dion, of the University of Lausanne in Switzerland.From nicking, “you end up with almost only contractions.” Suddenly, he realized, he had a potential therapeutic on his hands (Cinesi et al., 2016).The longer the repeat, the more room to place these obstacles, they reasoned.