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Amidst rising hopes for using CRISPR gene editing tools to repair deadly mutations linked to conditions like cystic fibrosis and sickle cell disease, a study in Communications Biology describes a new innovation that could accelerate this work by rapidly revealing unintended and potentially harmful changes introduced by a gene editing process.
"We've developed a new process for rapidly screening all of the edits made by CRISPR, and it shows there may be many more unintended changes to DNA around the site of a CRISPR repair than previously thought," said Eric Kmiec, Ph.D., director of ChristianaCare's Gene Editing Institute and the principle author of the study.
Study - Tool - Gene - Editing - Institute
The study describes a new tool developed at the Gene Editing Institute that in just 48 hours can identify "multiple outcomes of CRISPR-directed gene editing," a process that typically required up to two months of costly and complicated DNA analysis.
Kmiec cautioned that the unintended changes revealed by their work involve "subtle mutations" to DNA around the immediate site of the genome targeted for repair. That's very different, he said, from the hotly debated concern about the risk of CRISPR causing "off-target" mutations by drifting far afield from the intended site and making random cuts across the genome.
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"It's important to note that in all instances, we were still seeing CRISPR achieve a fantastic level of successful repairs that would have been unimaginable even five years ago," said lead author Brett Sansbury. "But we saw a lot of other changes to DNA near the site of the repair that need to be better understood so that when we correct one problem, we're not creating another."
She said those changes included deletions, duplications and rearrangements of DNA code. And while the researchers believe the vast majority of these unintended edits may have no consequence for patients, it's important to identify them...
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