SCIENTISTS have developed a new tool that will make it easier to eradicate inherited disease.
They hope the gene-editing therapy will allow them to repair DNA mutations.
Many common diseases such as genetic blindness, sickle-cell anaemia and cystic fibrosis are due to a single DNA fault.
Until now, scientists were using a technique called Crispr to cut out and replace defective genes.
But the experiments have also introduced errors that could cause unintended long-term consequences.
DNA is a code made up of four bases – A, G, C and T.
In tests, the new tool directly changed these building blocks to eradicate faults.
But it did not introduce mistakes.
Professor David Liu, from Harvard University, who led the US research, said: “We developed a new base editor, a molecular machine, that in a programmable, irreversible, efficient, and clean manner can correct these mutations in the genome of living cells.
“When targeted to certain sites in human genomic DNA, this conversion reverses the mutation that is associated with a particular disease.”
The results are reported in the journal Nature.
Dr Liu said a more work is needed before the technique could be used to help human patients.
British scientists called the research “exciting” and “incredibly powerful”.
Professor Robin Lovell-Badge, group leader at The Francis Crick Institute, London, said: “This is both clever and important science.”
And Dr Kosuke Yusa, Group Leader at the Wellcome Trust Sanger Institute, said: “The new editor reported in this study represents substantial therapeutic potential to possibly correct disease mutations in the future.”
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