
The CRISPR genome-editing method may just have become even more powerful. ’s team at McGill University in Canada has managed to repair mutations in 90 per cent of target cells using CRISPR – the best success rate yet.
The CRISPR approach is very good at disabling genes, but using the technique to fix them is much harder, because it involves replacing a faulty sequence with another. This typically works in less than 10 per cent of target cells.
To make the process more efficient, Akavia’s team physically linked the replacement DNA with the CRISPR protein that finds and cuts the faulty sequence. This ensures that the replacement DNA is there ready to be slotted in once the cut is made. “We’ve taped the [replacement] text to the scissors,” says Akavia.
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Targeting diseases
The team also used a polymer called polyethylenimine to target this protein-DNA complex directly to the nucleus of cells, so less goes to waste.
The highest efficiency rate reported by other teams exploring similar approaches so far is 60 per cent, so Akavia’s method could be a big step forward. It now needs to be tested in a wide variety of cells to confirm it is generally applicable.
If confirmed, the technique could be useful for treating or preventing essentially all inherited diseases, from cystic fibrosis to Huntington’s.
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