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posted by janrinok on Wednesday January 01 2020, @10:34AM   Printer-friendly
from the crispier dept.

Super-precise new CRISPR tool could tackle a plethora of genetic diseases:

For all the ease with which the wildly popular CRISPR–Cas9 gene-editing tool alters genomes, it's still somewhat clunky and prone to errors and unintended effects. Now, a recently developed alternative offers greater control over genome edits — an advance that could be particularly important for developing gene therapies.

The alternative method, called prime editing, improves the chances that researchers will end up with only the edits they want, instead of a mix of changes that they can't predict. The tool, described in a study published on 21 October in Nature, also reduces the 'off-target' effects that are a key challenge for some applications of the standard CRISPR–Cas9 system. That could make prime-editing-based gene therapies safer for use in people.

The tool also seems capable of making a wider variety of edits, which might one day allow it to be used to treat the many genetic diseases that have so far stymied gene-editors. David Liu, a chemical biologist at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts and lead study author, estimates that prime editing might help researchers tackle nearly 90% of the more than 75,000 disease-associated DNA variants listed in ClinVar, a public database developed by the US National Institutes of Health.

The specificity of the changes that this latest tool is capable of could also make it easier for researchers to develop models of disease in the laboratory, or to study the function of specific genes, says Liu.

"It's early days, but the initial results look fantastic," says Brittany Adamson, who studies DNA repair and gene editing at Princeton University in New Jersey. "You're going to see a lot of people using it."

Prime editing may not be able to make the very big DNA insertions or deletions that CRISPR–Cas9 is capable of — so it's unlikely to completely replace the well-established editing tool, says molecular biologist Erik Sontheimer at the University of Massachusetts Medical School in Worcester. That's because for prime editing, the change that a researcher wants to make is encoded on a strand of RNA. The longer that strand gets, the more likely it is to be damaged by enzymes in the cell.

"Different flavours of genome-editing platforms are still going to be needed for different types of edits," says Sontheimer.


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  • (Score: 2) by Freeman on Thursday January 02 2020, @04:40PM

    by Freeman (732) on Thursday January 02 2020, @04:40PM (#938666) Journal

    Yeah, that dystopian future is quite a ways in the future and relies on a lot of variables going horribly wrong in just the right way.

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