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A new CRISPR system can switch single letters of the genome efficiently, in a way that could reliably repair many disease-causing mutations.


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The most clever CRISPR gadget to date!

all the hoopla about CRISPR, the revolutionary genome-editing technology has a dirty little secret: it’s a very messy business. Scientists basically whack the famed double helix with a molecular machete, often triggering the cell’s DNA repair machinery to make all sorts of unwanted changes to the genome beyond what they intended.

On Wednesday, researchers unveiled in Nature a significant improvement — a new CRISPR system that can switch single letters of the genome cleanly and efficiently, in a way that they say could reliably repair many disease-causing mutations.

Because of “the cell’s desperate attempts” to mend its genome, said Harvard University biologist George Church, “what often passes as ‘genome editing’ would more appropriately be called ‘genome vandalism,’” as the cell inserts and deletes random bits of DNA where CRISPR cuts it.

Because the new version of CRISPR avoids that mess, it “offers a huge step forward,” said Church, who was not involved in the discovery, and whose 2013 paper helped launch the CRISPR frenzy. “It is arguably the most clever CRISPR gadget to date.”

If the CRISPR invention lives up to its promise — the experiments were done in cells in lab dishes, not whole animals or people — it could fix misspellings in DNA that cause Tay-Sachs, neurofibromatosis, sickle cell, cystic fibrosis, and other devastating inherited diseases, as well as mutations that raise the risk of diseases as common as cancer and Alzheimer’s.

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